363 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2023 Canarutto D, Asperti C, Vavassori V, Porcellini S, Rovelli E, Paulis M, Ferrari S, Varesi A, Fiumara M, Jacob A, Sergi Sergi L, Visigalli I, Ferrua F, González-Granado LI, Lougaris V, ... ... Naldini L, et al. Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4 T-cells for the treatment of Hyper IgM1. The Embo Journal. e114188. PMID 37916874 DOI: 10.15252/embj.2023114188  0.351
2023 Kerzel T, Giacca G, Beretta S, Bresesti C, Notaro M, Scotti GM, Balestrieri C, Canu T, Redegalli M, Pedica F, Genua M, Ostuni R, Kajaste-Rudnitski A, Oshima M, Tonon G, ... ... Naldini L, et al. In vivo macrophage engineering reshapes the tumor microenvironment leading to eradication of liver metastases. Cancer Cell. 41: 1892-1910.e10. PMID 37863068 DOI: 10.1016/j.ccell.2023.09.014  0.303
2023 Asperti C, Canarutto D, Porcellini S, Sanvito F, Cecere F, Vavassori V, Ferrari S, Rovelli E, Albano L, Jacob A, Sergi Sergi L, Montaldo E, Ferrua F, González-Granado LI, Lougaris V, ... ... Naldini L, et al. Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated and . Molecular Therapy. Methods & Clinical Development. 30: 546-557. PMID 37693944 DOI: 10.1016/j.omtm.2023.08.020  0.353
2023 Ferrari S, Naldini L. A step toward stem cell engineering in vivo. Science (New York, N.Y.). 381: 378-379. PMID 37499013 DOI: 10.1126/science.adj0997  0.308
2023 Canarutto D, Omer Javed A, Pedrazzani G, Ferrari S, Naldini L. Mobilization-based engraftment of haematopoietic stem cells: a new perspective for chemotherapy-free gene therapy and transplantation. British Medical Bulletin. PMID 37460391 DOI: 10.1093/bmb/ldad017  0.335
2023 Vavassori V, Ferrari S, Beretta S, Asperti C, Albano L, Annoni A, Gaddoni C, Varesi A, Soldi M, Cuomo A, Bonaldi T, Radrizzani M, Merelli I, Naldini L. Lipid Nanoparticles Allow Efficient and Harmless Ex Vivo Gene Editing of Human Hematopoietic Cells. Blood. PMID 37294917 DOI: 10.1182/blood.2022019333  0.354
2023 Ferrari S, Valeri E, Conti A, Scala S, Aprile A, Di Micco R, Kajaste-Rudnitski A, Montini E, Ferrari G, Aiuti A, Naldini L. Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy. Cell Stem Cell. 30: 549-570. PMID 37146580 DOI: 10.1016/j.stem.2023.04.014  0.357
2022 Omer-Javed A, Pedrazzani G, Albano L, Ghaus S, Latroche C, Manzi M, Ferrari S, Fiumara M, Jacob A, Vavassori V, Nonis A, Canarutto D, Naldini L. Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells. Cell. 185: 2248-2264.e21. PMID 35617958 DOI: 10.1016/j.cell.2022.04.039  0.378
2022 Milani M, Canepari C, Liu T, Biffi M, Russo F, Plati T, Curto R, Patarroyo-White S, Drager D, Visigalli I, Brombin C, Albertini P, Follenzi A, Ayuso E, Mueller C, ... ... Naldini L, et al. Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates. Nature Communications. 13: 2454. PMID 35508619 DOI: 10.1038/s41467-022-30102-3  0.315
2022 Tucci F, Galimberti S, Naldini L, Valsecchi MG, Aiuti A. A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders. Nature Communications. 13: 1315. PMID 35288539 DOI: 10.1038/s41467-022-28762-2  0.303
2022 Naldini L, Cicalese MP, Bernardo ME, Gentner B, Gabaldo M, Ferrari G, Aiuti A. The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy. Hemasphere. 6: e671. PMID 35198856 DOI: 10.1097/HS9.0000000000000671  0.321
2021 Zoccolillo M, Brigida I, Barzaghi F, Scala S, Hernández RJ, Basso-Ricci L, Colantuoni M, Pettinato E, Sergi LS, Milardi G, Capasso P, Lombardo A, Gregori S, Sanvito F, Schena F, ... ... Naldini L, et al. Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency. Blood Advances. 5: 3174-3187. PMID 34424322 DOI: 10.1182/bloodadvances.2020003811  0.344
2021 Cesana D, Calabria A, Rudilosso L, Gallina P, Benedicenti F, Spinozzi G, Schiroli G, Magnani A, Acquati S, Fumagalli F, Calbi V, Witzel M, Bushman FD, Cantore A, Genovese P, ... ... Naldini L, et al. Retrieval of vector integration sites from cell-free DNA. Nature Medicine. PMID 34140705 DOI: 10.1038/s41591-021-01389-4  0.323
2021 Ferrari S, Beretta S, Jacob A, Cittaro D, Albano L, Merelli I, Naldini L, Genovese P. BAR-Seq clonal tracking of gene-edited cells. Nature Protocols. 16: 2991-3025. PMID 34031609 DOI: 10.1038/s41596-021-00529-x  0.32
2021 Vavassori V, Mercuri E, Marcovecchio GE, Castiello MC, Schiroli G, Albano L, Margulies C, Buquicchio F, Fontana E, Beretta S, Merelli I, Cappelleri A, Rancoita PM, Lougaris V, Plebani A, ... ... Naldini L, et al. Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome. Embo Molecular Medicine. e13545. PMID 33475257 DOI: 10.15252/emmm.202013545  0.365
2021 Finocchiaro G, Gentner B, Farina F, Capotondo A, Eoli M, Anghileri E, Carabba MG, Cuccarini V, DI Meco F, Legnani F, Pollo B, Bruzzone MG, Saini M, Ferroli P, Pallini R, ... ... Naldini L, et al. A phase I-IIa study of genetically modified Tie-2 expressing monocytes in patients with glioblastoma multiforme (TEM-GBM Study). Journal of Clinical Oncology. 39: 2532-2532. DOI: 10.1200/JCO.2021.39.15_SUPPL.2532  0.324
2021 Mortellaro A, Zoccolillo M, Mesa Nuñez C, Brix A, Brigida I, Barzaghi F, Scala S, Jofra Hernández R, Basso-Ricci L, Colantuoni M, Cesaro S, Conti F, Pession A, Benedetti F, Gattorno M, ... Naldini L, et al. Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency Blood. 138: 2937-2937. DOI: 10.1182/blood-2021-152127  0.331
2021 Gentner B, Finocchiaro G, Farina F, Eoli M, Alessia C, Anghileri E, Antonarelli G, Barcella M, Bruzzone MG, Carrabba MG, Cuccarini V, D'Alessandris G, Di Meco F, Ferla V, Ferroli P, ... ... Naldini L, et al. TEM-GBM: An Open-Label, Phase I/IIa Dose-Escalation Study Evaluating the Safety and Efficacy of Genetically Modified Tie-2 Expressing Monocytes to Deliver IFN-α within Glioblastoma Tumor Microenvironment Blood. 138: 2844-2844. DOI: 10.1182/blood-2021-150230  0.301
2021 Vavassori V, Mercuri E, Marcovecchio G, Castiello MC, Canarutto D, Asperti C, Jacob A, Albano L, Fontana E, Scanziani E, Radrizzani M, Villa A, Genovese P, Naldini L. Towards Clinical Translation of Hematopoietic Cell Gene Editing for Treating Hyper-IgM Type 1 Blood. 138: 3978-3978. DOI: 10.1182/blood-2021-148572  0.395
2021 Fiumara M, Ferrari S, Omer A, Beretta S, Albano L, Merelli I, Naldini L. Assessing Stealth and Sensed Base Editing in Human Hematopoietic Stem/Progenitor Cells Blood. 138: 3976-3976. DOI: 10.1182/blood-2021-147700  0.325
2021 Gentner B, Bernardo ME, Tucci F, Fumagalli F, Pontesilli S, Silvani P, Zonari E, Miglietta S, Montini E, Ciceri F, La Marca G, Parini R, Naldini L, Aiuti A. Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome) Molecular Genetics and Metabolism. 132: S42-S43. DOI: 10.1016/J.YMGME.2020.12.087  0.329
2021 Fumagalli F, Calbi V, Sessa M, Zambon A, Fratini E, Cicalese MP, Gallo V, Ciotti F, Sarzana M, Facchini M, Locatelli S, Antonioli G, Zancan S, Segovia J, Schwab LC, ... ... Naldini L, et al. Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy Molecular Genetics and Metabolism. 132: S40. DOI: 10.1016/J.YMGME.2020.12.081  0.315
2021 Calbi V, Fumagalli F, De Mattia F, Fratini E, Ferrua F, Barzaghi F, Cicalese MP, Migliavacca M, Tucci F, Gallo V, Ciotti F, Fraschini M, Sarzana M, Locatelli S, Sangalli M, ... ... Naldini L, et al. Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200 Molecular Genetics and Metabolism. 132: S21-S22. DOI: 10.1016/J.YMGME.2020.12.031  0.337
2020 Soldi M, Sergi Sergi L, Unali G, Kerzel T, Cuccovillo I, Capasso P, Annoni A, Biffi M, Rancoita PMV, Cantore A, Lombardo A, Naldini L, Squadrito ML, Kajaste-Rudnitski A. Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced and Genetic Engineering. Molecular Therapy. Methods & Clinical Development. 19: 411-425. PMID 33294490 DOI: 10.1016/j.omtm.2020.10.009  0.311
2020 Jofra Hernández R, Calabria A, Sanvito F, De Mattia F, Farinelli G, Scala S, Visigalli I, Carriglio N, De Simone M, Vezzoli M, Cecere F, Migliavacca M, Basso-Ricci L, Omrani M, Benedicenti F, ... ... Naldini L, et al. Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 33010230 DOI: 10.1016/j.ymthe.2020.09.030  0.365
2020 Chanut F, Sanvito F, Ferrari G, Visigalli I, Carriglio N, Jofra Hernandez R, Norata R, Doglioni C, Naldini L, Cristofori P. Conditioning regimens in long term nonclinical studies to support development of ex-vivo gene therapy: review of non-proliferative and proliferative changes. Human Gene Therapy. PMID 32883113 DOI: 10.1089/Hum.2020.135  0.423
2020 Ferrari S, Jacob A, Beretta S, Unali G, Albano L, Vavassori V, Cittaro D, Lazarevic D, Brombin C, Cugnata F, Kajaste-Rudnitski A, Merelli I, Genovese P, Naldini L. Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking. Nature Biotechnology. PMID 32601433 DOI: 10.1038/S41587-020-0551-Y  0.471
2020 Cantore A, Naldini L. WFH State-of-the-art paper 2020: In vivo lentiviral vector gene therapy for haemophilia. Haemophilia : the Official Journal of the World Federation of Hemophilia. PMID 32537776 DOI: 10.1111/Hae.14056  0.48
2020 Birocchi F, Cusimano M, Ranghetti A, Tomaso TD, Costa B, Angel P, Coltella N, Naldini L. Abstract PR13: Development of chimeric forms of IFN-alpha for “on demand” in vivo cancer gene therapy Cancer Immunology Research. 8. DOI: 10.1158/2326-6074.Tumimm18-Pr13  0.388
2020 Fumagalli F, Calbi V, Sessa M, Zambon A, Baldoli C, Rancoita PMV, Acquati S, Mattia Fd, Tucci F, Gallo V, Zancan S, Montini E, Schwab L, Downey G, Sharpe J, ... ... Naldini L, et al. Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients Molecular Genetics and Metabolism. 129. DOI: 10.1016/J.Ymgme.2019.11.135  0.416
2019 Gentner B, Naldini L. In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease. Cell Stem Cell. 25: 592-593. PMID 31703769 DOI: 10.1016/J.Stem.2019.10.004  0.481
2019 Bénéchet AP, De Simone G, Di Lucia P, Cilenti F, Barbiera G, Le Bert N, Fumagalli V, Lusito E, Moalli F, Bianchessi V, Andreata F, Zordan P, Bono E, Giustini L, Bonilla WV, ... ... Naldini L, et al. Dynamics and genomic landscape of CD8 T cells undergoing hepatic priming. Nature. PMID 31582858 DOI: 10.1038/S41586-019-1620-6  0.362
2019 Bortolomai I, Sandri M, Draghici E, Fontana E, Campodoni E, Marcovecchio GE, Ferrua F, Perani L, Spinelli A, Canu T, Catucci M, Di Tomaso T, Sergi Sergi L, Esposito A, Lombardo A, ... Naldini L, et al. Gene Modification and Three-Dimensional Scaffolds as Novel Tools to Allow the Use of Postnatal Thymic Epithelial Cells for Thymus Regeneration Approaches. Stem Cells Translational Medicine. PMID 31140762 DOI: 10.1002/Sctm.18-0218  0.447
2019 Milani M, Annoni A, Moalli F, Liu T, Cesana D, Calabria A, Bartolaccini S, Biffi M, Russo F, Visigalli I, Raimondi A, Patarroyo-White S, Drager D, Cristofori P, Ayuso E, ... ... Naldini L, et al. Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates. Science Translational Medicine. 11. PMID 31118293 DOI: 10.1126/Scitranslmed.Aav7325  0.446
2019 Squeri G, Passerini L, Ferro F, Laudisa C, Tomasoni D, Deodato F, Donati MA, Gasperini S, Aiuti A, Bernardo ME, Gentner B, Naldini L, Annoni A, Biffi A, Gregori S. Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 31060789 DOI: 10.1016/J.Ymthe.2019.04.014  0.469
2019 Petrillo C, Calabria A, Piras F, Capotondo A, Spinozzi G, Cuccovillo I, Benedicenti F, Naldini L, Montini E, Biffi A, Gentner B, Kajaste-Rudnitski A. Assessing the impact of Cyclosporine A on lentiviral transduction and preservation of human hematopoietic stem cells in clinically relevant ex-vivo gene therapy settings. Human Gene Therapy. PMID 31037976 DOI: 10.1089/Hum.2019.016  0.508
2019 Ferrua F, Cicalese MP, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo ME, Calbi V, Assanelli AA, Facchini M, Fossati C, Albertazzi E, Scaramuzza S, Brigida I, ... ... Naldini L, et al. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. The Lancet. Haematology. PMID 30981783 DOI: 10.1016/S2352-3026(19)30021-3  0.45
2019 Schiroli G, Conti A, Ferrari S, Della Volpe L, Jacob A, Albano L, Beretta S, Calabria A, Vavassori V, Gasparini P, Salataj E, Ndiaye-Lobry D, Brombin C, Chaumeil J, Montini E, ... ... Naldini L, et al. Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response. Cell Stem Cell. PMID 30905619 DOI: 10.1016/J.Stem.2019.02.019  0.384
2019 Marktel S, Scaramuzza S, Cicalese MP, Giglio F, Galimberti S, Lidonnici MR, Calbi V, Assanelli A, Bernardo ME, Rossi C, Calabria A, Milani R, Gattillo S, Benedicenti F, Spinozzi G, ... ... Naldini L, et al. Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia. Nature Medicine. PMID 30664781 DOI: 10.1038/S41591-018-0301-6  0.449
2019 Gentner B, Bernardo ME, Tucci F, Zonari E, Fumagalli F, Pontesilli S, Acquati S, Silvani P, Ciceri F, Rovelli A, Marca GL, Parini R, Gregori S, Montini E, Naldini L, et al. Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial Blood. 134: 607-607. DOI: 10.1182/Blood-2019-128805  0.386
2019 Ferrua F, Cicalese MP, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo ME, Calbi V, Tucci F, Assanelli AA, Peccatori J, Albertazzi E, Clerici AG, Salerio FA, ... ... Naldini L, et al. Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010 Blood. 134: 3346-3346. DOI: 10.1182/Blood-2019-124665  0.407
2019 Gentner B, Farina F, Carrabba MG, Marcatti M, Girlanda S, Antonarelli G, Alessia C, Mazzoleni S, Russo C, Naldini L, Ciceri F. TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy Blood. 134: 2064-2064. DOI: 10.1182/Blood-2019-123749  0.351
2019 Finocchiaro G, Gentner B, Ciceri F, DiMeco F, Legnani F, Eoli M, Pollo B, Farina F, Zambanini A, Mazzoleni S, Russo C, Naldini L. ATIM-36. TEM-GBM-001 STUDY: AUTOLOGOUS CD34+ ENRICHED HEMATOPOIETIC PROGENITOR CELLS GENETICALLY MODIFIED FOR HUMAN INTERFERON-α2 & ADMINISTERED TO PATIENTS WITH GLIOBLASTOMA & AN UNMETHYLATED MGMT PROMOTER Neuro-Oncology. 21. DOI: 10.1093/Neuonc/Noz175.035  0.344
2019 Gentner B, Ciceri F, DiMeco F, Legnani F, Eoli M, Pollo B, Farina F, Mazzoleni S, Russo C, Naldini L, Finocchiaro G. P05.02 A phase I/IIa dose escalation study evaluating the safety and efficacy of autologous CD34+ enriched hematopoietic progenitor cells genetically modified for human interferon-α2 in patients with GBM and an unmethylated MGMT promoter (TEM-GBM-001) Neuro-Oncology. 21. DOI: 10.1093/Neuonc/Noz126.116  0.323
2018 Petrillo C, Thorne LG, Unali G, Schiroli G, Giordano AMS, Piras F, Cuccovillo I, Petit SJ, Ahsan F, Noursadeghi M, Clare S, Genovese P, Gentner B, Naldini L, Towers GJ, et al. Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells. Cell Stem Cell. PMID 30416070 DOI: 10.1016/J.Stem.2018.10.008  0.483
2018 Lidonnici MR, Paleari Y, Tiboni F, Mandelli G, Rossi C, Vezzoli M, Aprile A, Lederer CW, Ambrosi A, Chanut F, Sanvito F, Calabria A, Poletti V, Mavilio F, Montini E, ... Naldini L, et al. Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia. Molecular Therapy. Methods & Clinical Development. 11: 9-28. PMID 30320151 DOI: 10.1016/J.Omtm.2018.09.001  0.468
2018 Escobar G, Barbarossa L, Barbiera G, Norelli M, Genua M, Ranghetti A, Plati T, Camisa B, Brombin C, Cittaro D, Annoni A, Bondanza A, Ostuni R, Gentner B, Naldini L. Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens. Nature Communications. 9: 2896. PMID 30042420 DOI: 10.1038/S41467-018-05315-0  0.48
2018 Annoni A, Gregori S, Naldini L, Cantore A. Modulation of immune responses in lentiviral vector-mediated gene transfer. Cellular Immunology. PMID 29735164 DOI: 10.1016/J.Cellimm.2018.04.012  0.438
2018 Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Marcella F, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, ... ... Naldini L, et al. Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy. Bone Marrow Transplantation. PMID 29379168 DOI: 10.1038/S41409-017-0085-1  0.374
2018 Cianciotti BC, Potenza A, Vavassori V, Magnani Z, Naldini L, Ciceri F, Genovese P, Ruggiero E, Bonini C. Generation of Memory Stem T Cells Specific for Tumor Antigens and Resistant to Inhibitory Signals By Genome Editing Blood. 132: 2202-2202. DOI: 10.1182/Blood-2018-99-118201  0.31
2017 Benedetti S, Uno N, Hoshiya H, Ragazzi M, Ferrari G, Kazuki Y, Moyle LA, Tonlorenzi R, Lombardo A, Chaouch S, Mouly V, Moore M, Popplewell L, Kazuki K, Katoh M, ... Naldini L, et al. Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy. Embo Molecular Medicine. PMID 29242210 DOI: 10.15252/emmm.201607284  0.37
2017 Benedetti S, Uno N, Hoshiya H, Ragazzi M, Ferrari G, Kazuki Y, Moyle LA, Tonlorenzi R, Lombardo A, Chaouch S, Mouly V, Moore M, Popplewell L, Kazuki K, Katoh M, ... Naldini L, et al. Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy. Embo Molecular Medicine. PMID 29242210 DOI: 10.15252/emmm.201607284  0.37
2017 Sio FRSd, Passerini L, Valente MM, Russo F, Naldini L, Roncarolo MG, Bacchetta R. Ectopic FOXP3 Expression Preserves Primitive Features Of Human Hematopoietic Stem Cells While Impairing Functional T Cell Differentiation. Scientific Reports. 7: 15820-15820. PMID 29150659 DOI: 10.1038/S41598-017-15689-8  0.499
2017 Schiroli G, Ferrari S, Conway A, Jacob A, Capo V, Albano L, Plati T, Castiello MC, Sanvito F, Gennery AR, Bovolenta C, Palchaudhuri R, Scadden DT, Holmes MC, Villa A, ... ... Naldini L, et al. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. Science Translational Medicine. 9. PMID 29021165 DOI: 10.1126/Scitranslmed.Aan0820  0.469
2017 Diez B, Genovese P, Roman-Rodriguez FJ, Alvarez L, Schiroli G, Ugalde L, Rodriguez-Perales S, Sevilla J, Diaz de Heredia C, Holmes MC, Lombardo A, Naldini L, Bueren JA, Rio P. Therapeutic gene editing in CD34(+) hematopoietic progenitors from Fanconi anemia patients. Embo Molecular Medicine. PMID 28899930 DOI: 10.15252/Emmm.201707540  0.496
2017 Milani M, Annoni A, Bartolaccini S, Biffi M, Russo F, Di Tomaso T, Raimondi A, Lengler J, Holmes MC, Scheiflinger F, Lombardo A, Cantore A, Naldini L. Genome editing for scalable production of alloantigen-free lentiviral vectors for in vivo gene therapy. Embo Molecular Medicine. PMID 28835507 DOI: 10.15252/Emmm.201708148  0.475
2017 Piras F, Riba M, Petrillo C, Lazarevic D, Cuccovillo I, Bartolaccini S, Stupka E, Gentner B, Cittaro D, Naldini L, Kajaste-Rudnitski A. Lentiviral vectors escape innate sensing but trigger p53 in human hematopoietic stem and progenitor cells. Embo Molecular Medicine. PMID 28667090 DOI: 10.15252/Emmm.201707922  0.489
2017 Mastaglio S, Genovese P, Magnani Z, Ruggiero E, Landoni E, Camisa B, Schiroli G, Provasi E, Lombardo A, Reik A, Cieri N, Rocchi M, Oliveira G, Escobar G, Casucci M, ... ... Naldini L, et al. NY-ESO-1 TCR single edited central memory and memory stem T cells to treat multiple myeloma without inducing GvHD. Blood. PMID 28637663 DOI: 10.1182/Blood-2016-08-732636  0.469
2017 Aiuti A, Roncarolo MG, Naldini L. Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products. Embo Molecular Medicine. PMID 28396566 DOI: 10.15252/Emmm.201707573  0.455
2017 Zonari E, Desantis G, Petrillo C, Boccalatte FE, Lidonnici MR, Kajaste-Rudnitski A, Aiuti A, Ferrari G, Naldini L, Gentner B. Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy. Stem Cell Reports. PMID 28330619 DOI: 10.1016/J.Stemcr.2017.02.010  0.485
2017 Cantore A, Milani M, Annoni A, Liu T, Bartolaccini S, Biffi M, Russo F, Peters R, Lombardo A, Nichols TC, Ayuso E, Naldini L. Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors Blood. 130: 605-605. DOI: 10.1182/Blood.V130.Suppl_1.605.605  0.477
2017 Marktel S, Cicalese MP, Giglio F, Scaramuzza S, Calbi V, Casiraghi M, Ciotti F, Lidonnici MR, Rossi C, Masera N, D'Angelo E, Mirra N, Origa R, Tartaglione I, Mandelli G, ... ... Naldini L, et al. Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector Blood. 130: 355-355. DOI: 10.1182/Blood.V130.Suppl_1.355.355  0.453
2017 Fumagalli F, Calbi V, Zambon A, Ciotti F, Lorioli L, Sessa M, Sarzana M, Canale S, Antonioli G, Medaglini S, Del Carro U, Quattrini A, Baldoli C, Martino S, Di Serio C, ... ... Naldini L, et al. Update on safety and efficacy of lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD) European Journal of Paediatric Neurology. 21: e20. DOI: 10.1016/J.EJPN.2017.04.986  0.315
2016 Naldini L, Parker GC, Flotte TR. The Renaissance of Gene and Cell Therapy: Florence 2016. Human Gene Therapy. 27: 727-728. PMID 29112477 DOI: 10.1089/Hum.2016.29034.Lna  0.453
2016 Amabile A, Migliara A, Capasso P, Biffi M, Cittaro D, Naldini L, Lombardo A. Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing. Cell. 167: 219-232.e14. PMID 27662090 DOI: 10.1016/J.Cell.2016.09.006  0.422
2016 Naldini L, Trono D, Verma IM. Lentiviral vectors, two decades later. Science (New York, N.Y.). 353: 1101-2. PMID 27609877 DOI: 10.1126/Science.Aah6192  0.617
2016 Cartier-Lacave N, Ali R, Ylä-Herttuala S, Kato K, Baetschi B, Lovell-Badge R, Naldini L, Thrasher A. Debate on Germline Gene Editing. Human Gene Therapy Methods. 27: 135-42. PMID 27529507 DOI: 10.1089/Hgtb.2016.28999.Deb  0.38
2016 Cottone L, Capobianco A, Gualteroni C, Monno A, Raccagni I, Valtorta S, Canu T, Tomaso TD, Lombardo A, Esposito A, Moresco RM, Maschio AD, Naldini L, Rovere-Querini P, Bianchi ME, et al. Leukocytes recruited by tumor-derived HMGB1 sustain peritoneal carcinomatosis. Oncoimmunology. 5: e1122860. PMID 27467932 DOI: 10.1080/2162402X.2015.1122860  0.323
2016 Visigalli I, Delai S, Ferro F, Cecere F, Vezzoli M, Sanvito F, Chanut F, Benedicenti F, Spinozzi G, Wynn R, Calabria A, Naldini L, Montini E, Cristofori P, Biffi A. Preclinical testing of the safety and tolerability of LV-mediated above normal alpha-L-iduronidase expression in murine and human hematopoietic cells using toxicology and biodistribution GLP studies. Human Gene Therapy. PMID 27431943 DOI: 10.1089/Hum.2016.068  0.501
2016 Aiuti A, Naldini L. Safer conditioning for blood stem cell transplants. Nature Biotechnology. 34: 721-3. PMID 27404882 DOI: 10.1038/Nbt.3629  0.405
2016 Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae C, Vicenzi E, Finocchi A, Bredius RGM, ... ... Naldini L, et al. Erratum: Preclinical Safety and Efficacy of Human CD34+ Cells Transduced with Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome (Molecular Therapy (2013) 21 (175-184) DOI:10.1038/mt.2012.23) Molecular Therapy. 24. PMID 27324447 DOI: 10.1038/Mt.2016.96  0.363
2016 Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, ... ... Naldini L, et al. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet (London, England). PMID 27289174 DOI: 10.1016/S0140-6736(16)30374-9  0.421
2016 Biasco L, Pellin D, Scala S, Dionisio F, Basso-Ricci L, Leonardelli L, Scaramuzza S, Baricordi C, Ferrua F, Cicalese MP, Giannelli S, Neduva V, Dow DJ, Schmidt M, Von Kalle C, ... ... Naldini L, et al. In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases. Cell Stem Cell. PMID 27237736 DOI: 10.1016/J.Stem.2016.04.016  0.405
2016 Meneghini V, Lattanzi A, Tiradani L, Bravo G, Morena F, Sanvito F, Calabria A, Bringas J, Fisher-Perkins JM, Dufour JP, Baker KC, Doglioni C, Montini E, Bunnell BA, Bankievicz K, ... ... Naldini L, et al. Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy. Embo Molecular Medicine. PMID 27025653 DOI: 10.15252/Emmm.201505850  0.439
2016 Lechman ER, Gentner B, Ng SW, Schoof EM, van Galen P, Kennedy JA, Nucera S, Ciceri F, Kaufmann KB, Takayama N, Dobson SM, Trotman-Grant A, Krivdova G, Elzinga J, Mitchell A, ... ... Naldini L, et al. miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells. Cancer Cell. PMID 26832662 DOI: 10.1016/J.Ccell.2015.12.011  0.312
2016 Catarinella M, Monestiroli A, Escobar G, Fiocchi A, Tran NL, Aiolfi R, Marra P, Esposito A, Cipriani F, Aldrighetti L, Iannacone M, Naldini L, Guidotti LG, Sitia G. IFNα gene/cell therapy curbs colorectal cancer colonization of the liver by acting on the hepatic microenvironment. Embo Molecular Medicine. PMID 26769348 DOI: 10.15252/Emmm.201505395  0.394
2016 Zonari E, Desantis G, Petrillo C, Meo O, Scaramuzza S, Lidonnici MR, Kajaste-Rudnitski A, Ciceri F, Marktel S, Aiuti A, Ferrari G, Naldini L, Gentner B. Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications Blood. 128: 4707-4707. DOI: 10.1182/Blood.V128.22.4707.4707  0.363
2016 Escobar G, Moi D, Galea R, Sherif M, Ranghetti A, Naldini L, Mazzieri R. Abstract B181: Genetic engineering of tumor-infiltrating monocytes to inhibit primary and metastatic breast cancer Cancer Immunology Research. 4. DOI: 10.1158/2326-6074.Cricimteatiaacr15-B181  0.376
2016 Naldini L, Parker GC, Flotte TR. The Renaissance of Gene and Cell Therapy: Florence 2016 Human Gene Therapy. 27: 727-728. DOI: 10.1089/hum.2016.29034.lna  0.347
2016 Cantore A, Milani M, Liu T, Valle PD, D'Angelo A, VandenDriessche T, Chuah M, Jiang H, Nichols T, Naldini L. 756. Stable Amelioration of Hemophilia B in Dogs by Intravenous Administration of Lentiviral Vectors Expressing Hyper-Functional Factor IX Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33564-X  0.427
2016 Mastaglio S, Genovese P, Magnani Z, Landoni E, Camisa B, Schiroli G, Provasi E, Lombardo A, Reik A, Cieri N, Rocchi M, Oliveira G, Escobar G, Casucci M, Gentner B, ... ... Naldini L, et al. 752. Single Chain TCR Gene Editing in Adoptive Cell Therapy for Multiple Myeloma Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33560-2  0.433
2016 Amabile A, Migliara A, Capasso P, Biffi M, Naldini L, Lombardo A. 729. Inheritable Silencing of Endogenous Gene by Hit-and-Run Targeted Epigenetic Editing Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33537-7  0.457
2016 Díez B, Genovesse P, Schiroli G, Álvarez L, Román-Rodriguez FJ, Gregory PD, Holmes M, Lombardo A, Naldini L, Rio P, Bueren JA. 558. Targeted Gene Therapy in CD34+ Cells from Healthy Donors and Fanconi Anemia Patients Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33366-4  0.493
2016 Norelli M, Casucci M, Camisa B, Falcone L, Saudemont A, Naldini L, Ciceri F, Bordignon C, Bonini C, Bondanza A. 512. The Cytokine Release Syndrome Crucially Contributes to the Anti-Leukemic Effects of CD44v6 CAR-T Cells Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33321-4  0.4
2016 Cantore A, Milani M, Annoni A, Lengler J, Bartolaccini S, Tomaso TD, Holmes MC, Scheiflinger F, Lombardo A, Naldini L. 286. Genome Editing of Inducible Cell Lines for Scalable Production of Improved Lentiviral Vectors for Human Gene Therapy Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33095-7  0.499
2016 Castiello MC, Pala F, Morbach H, Schickel J, Scaramuzza S, Chamberlain N, Romberg N, Ferrua F, Candotti F, Burg Mvd, Naldini L, Aiuti A, Meffre E, Bosticardo M, Villa A. 280. Lentiviral-Mediated Gene Therapy Restores B Cell Homeostasis and Tolerance in Wiskott-Aldrich Syndrome Patients Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33089-1  0.402
2016 Zonari E, Meo O, Scaramuzza S, Montini E, Ferrari G, Naldini L, Gentner B. 235. Improved Ex Vivo Gene Therapy Using Highly Purified Hematopoietic Stem and Progenitor Cells Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33044-1  0.338
2016 Giuliani E, Piovan C, Bossi S, Corna S, Scavullo C, Pema M, Tomaso TD, Genovese P, Lombardo A, Naldini L, Bordignon C, Rizzardi GP, Bovolenta C. 130. Purification of Large Scale mRNA Encoding ZFN Nucleases by dHPLC Technology Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)32939-2  0.346
2016 Schiroli G, Genovese P, Capo V, Castiello MC, Albano L, Holmes MC, Villa A, Sitia G, Lombardo A, Naldini L. 42. Correction of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in the Mouse Model Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)32851-9  0.524
2016 Genovese P, Schiroli G, Conway A, Ferrari S, Guschin DY, Di Tomaso T, Albano L, Marin V, Giuliani E, Gregory PD, Holmes MC, Gentner B, Bovolenta C, Cost GJ, Lombardo A, ... Naldini L, et al. 37. Towards Clinical Translation of Hematopoietic Stem Cell Gene Editing for the Correction of SCID-X1 Mutations Molecular Therapy. 24: S16-S17. DOI: 10.1016/S1525-0016(16)32846-5  0.504
2015 Ungari S, Montepeloso A, Morena F, Cocchiarella F, Recchia A, Martino S, Gentner B, Naldini L, Biffi A. Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy. Molecular Therapy. Methods & Clinical Development. 2: 15038. PMID 26509184 DOI: 10.1038/Mtm.2015.38  0.505
2015 Naldini L. Gene therapy returns to centre stage. Nature. 526: 351-60. PMID 26469046 DOI: 10.1038/Nature15818  0.465
2015 Neri T, Muggeo S, Paulis M, Caldana ME, Crisafulli L, Strina D, Focarelli ML, Faggioli F, Recordati C, Scaramuzza S, Scanziani E, Mantero S, Buracchi C, Sobacchi C, Lombardo A, ... Naldini L, et al. Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts. Stem Cell Reports. 5: 558-68. PMID 26344905 DOI: 10.1016/J.Stemcr.2015.08.005  0.469
2015 Akbarpour M, Goudy KS, Cantore A, Russo F, Sanvito F, Naldini L, Annoni A, Roncarolo MG. Insulin B chain 9-23 gene transfer to hepatocytes protects from type 1 diabetes by inducing Ag-specific FoxP3+ Tregs. Science Translational Medicine. 7: 289ra81. PMID 26019217 DOI: 10.1126/Scitranslmed.Aaa3032  0.396
2015 Kajaste-Rudnitski A, Naldini L. Cellular innate immunity and restriction of viral infection: implications for lentiviral gene therapy in human hematopoietic cells. Human Gene Therapy. 26: 201-9. PMID 25808164 DOI: 10.1089/Hum.2015.036  0.521
2015 Castiello MC, Scaramuzza S, Pala F, Ferrua F, Uva P, Brigida I, Sereni L, van der Burg M, Ottaviano G, Albert MH, Roncarolo MG, Naldini L, Aiuti A, Villa A, Bosticardo M. B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome. The Journal of Allergy and Clinical Immunology. PMID 25792466 DOI: 10.1016/J.Jaci.2015.01.035  0.439
2015 Cesani M, Plati T, Lorioli L, Benedicenti F, Redaelli D, Dionisio F, Biasco L, Montini E, Naldini L, Biffi A. Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting. Gene Therapy. 22: 496-502. PMID 25762283 DOI: 10.1038/Gt.2015.10  0.509
2015 Cantore A, Ranzani M, Bartholomae CC, Volpin M, Valle PD, Sanvito F, Sergi LS, Gallina P, Benedicenti F, Bellinger D, Raymer R, Merricks E, Bellintani F, Martin S, Doglioni C, ... ... Naldini L, et al. Liver-directed lentiviral gene therapy in a dog model of hemophilia B. Science Translational Medicine. 7: 277ra28. PMID 25739762 DOI: 10.1126/Scitranslmed.Aaa1405  0.435
2015 Crivello P, Zito L, Sizzano F, Zino E, Maiers M, Mulder A, Toffalori C, Naldini L, Ciceri F, Vago L, Fleischhauer K. The impact of amino acid variability on alloreactivity defines a functional distance predictive of permissive HLA-DPB1 mismatches in hematopoietic stem cell transplantation. Biology of Blood and Marrow Transplantation : Journal of the American Society For Blood and Marrow Transplantation. 21: 233-41. PMID 25445022 DOI: 10.1016/J.Bbmt.2014.10.017  0.338
2015 Petrillo C, Cesana D, Piras F, Bartolaccini S, Naldini L, Montini E, Kajaste-Rudnitski A. Cyclosporin a and rapamycin relieve distinct lentiviral restriction blocks in hematopoietic stem and progenitor cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 352-62. PMID 25270076 DOI: 10.1038/Mt.2014.193  0.46
2015 Ferrua F, Cicalese MP, Galimberti S, Scaramuzza S, Giannelli S, Pajno R, Dionisio F, Biasco L, Castiello MC, Casiraghi M, Facchini M, Finocchi A, Metin A, Orange JS, Albert MH, ... ... Naldini L, et al. Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome Blood. 126: 259-259. DOI: 10.1182/Blood.V126.23.259.259  0.406
2015 Norelli M, Casucci M, Camisa B, Falcone L, Saudemont A, Genovese P, Naldini L, Bonini C, Bondanza A. 716. Durable Acute Myeloid Leukemia Remission Without Myeloablation in an Innovative Xenotolerant Mouse Model of CD44v6 CAR-T Cell Immunotherapy Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)34325-8  0.405
2015 Amabile A, Migliara A, Tomaso TD, Biffi M, Naldini L, Lombardo A. 690. Permanent Epigenetic Silencing of Human Genes With Artificial Transcriptional Repressors Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)34299-X  0.469
2015 Conway A, Guschin DY, Genovese P, Lombardo A, Naldini L, Gregory PD, Holmes MC, Cost GJ. 686. Gene Correction of IL2RG in Human Hematopoietic Stem and Progenitor Cells Molecular Therapy. 23: S273. DOI: 10.1016/S1525-0016(16)34295-2  0.506
2015 Schiroli G, Genovese P, Castiello MC, Capo V, Gregory PD, Holmes MC, Sitia G, Villa A, Lombardo A, Naldini L. 481. Targeted Genome Editing in Mouse Hematopoietic Stem/Progenitor Cells (HSPC) To Model Gene Correction of SCID-X1 Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)34090-4  0.531
2015 Biasco L, Dionisio F, Pellin D, Scala S, Ricci LB, Scaramuzza S, Baricordi C, Calabria A, Giannelli S, Neduva V, Dow DJ, Montini E, Serio CD, Naldini L, Aiuti A. 476. Clonal Tracking of Engineered Hematopoiesis In Vivo in Humans By Insertional Barcoding Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)34085-0  0.461
2015 Zonari E, Boccalatte F, Plati T, Aiuti A, Ferrari G, Montini E, Naldini L, Gentner B. 295. Hematopoietic Stem Cell Gene Therapy (2.0) Based on Purified CD34+CD38- Cells Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33904-1  0.408
2015 Escobar G, Ranghetti A, Barbarossa L, Zonari E, Nucera S, Ciceri F, Gentner B, Naldini L. 281. Engineering Hematopoiesis for Tumor-Targeted Interferon-alpha Delivery Inhibits Multuple Myeloma and B Cell Malignancies Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33890-4  0.515
2015 Genovese P, Mastaglio S, Magnani Z, Landoni E, Camisa B, Schiroli G, Provasi E, Lombardo A, Reik A, Cieri N, Ponzoni M, Ciceri F, Bordignon C, Holmes MC, Gregory PD, ... Naldini L, et al. 209. TCR Gene Editing in a Single Step of T Cell Activation To Redirect T Cell Specificity and Prevent GvHD Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33814-X  0.444
2015 Annoni A, Russo F, Cantore A, Naldini L, Roncarolo MG. 173. Insulin B9-23 LV-Driven Expression in Hepatocytes Combined With Suboptimal Dose of Anti-CD3 mAb Cures Type 1 Diabetes in NOD Mice Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33778-9  0.333
2015 Piras F, Riba M, Lazarevic D, Bartolaccini S, Stupka E, Cittaro D, Naldini L, Kajaste-Rudnitski A. 36. Genome-Wide Insight Into the Transcriptional Modulations Triggered By Lentiviral Transduction in Human Hematopoietic Stem Cells Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33640-1  0.435
2015 Cantore A, Milani M, Liu T, Valle PD, D'Angelo A, VandenDriessche T, Chuah M, Jiang H, Nichols T, Naldini L. 28. Intravenous Administration of Lentiviral Vectors Expressing Hyperactive Factor IX Converts Severe Into Mild Hemophilia B in a Canine Model Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33632-2  0.429
2015 Nucera S, Boccalatte F, Calabria A, Plati T, Fanciullo C, Manteiga J, Benedicenti F, Ciceri F, Ponzoni M, Montini E, Naldini L, Gentner B. 27. Aberrant Expression of the Stem Cell microRNA-126 Induces B Cell Malignancy Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33631-0  0.331
2015 Cantore A, Milani M, Lengler J, Bartolaccini S, Tomaso TD, Gregory PD, Scheiflinger F, Lombardo A, Naldini L. 6. Targeted Genome Editing of Cell Lines for Improved and Scalable Production of Lentiviral Vectors for Human Gene Therapy Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33610-3  0.496
2015 Volpin M, Calabria A, Cesana D, Tenderini E, Benedicenti F, Mitra K, Cohen-Haguenauer O, Naldini L, Lenz J, Stamatoyannopoulos G, Eugenio E. 3. Safety Assessment of SIN LVs Harboring Chromatin Insulators in the Sensitive Cdkn2a-/- In Vivo Genotoxicity Assay Show Enhancer-Blocking Activity of Specific Insulator Sequences Molecular Therapy. 23: S2. DOI: 10.1016/S1525-0016(16)33607-3  0.374
2015 Benedetti S, Hoshiya H, Ragazzi M, Uno N, Kazuki Y, Ferrari G, Tonlorenzi R, Lombardo A, Mouly V, Naldini L, Messina G, Oshimura M, Cossu G, Tedesco F. Reversible immortalization allows human artificial chromosome-mediated gene correction of human dystrophic muscle progenitor cells Neuromuscular Disorders. 25. DOI: 10.1016/J.Nmd.2015.06.243  0.354
2014 Lombardo A, Naldini L. Genome editing: a tool for research and therapy: targeted genome editing hits the clinic. Nature Medicine. 20: 1101-3. PMID 25295939 DOI: 10.1038/Nm.3721  0.347
2014 Ranzani M, Annunziato S, Calabria A, Brasca S, Benedicenti F, Gallina P, Naldini L, Montini E. Lentiviral vector-based insertional mutagenesis identifies genes involved in the resistance to targeted anticancer therapies. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 2056-68. PMID 25195596 DOI: 10.1038/Mt.2014.174  0.392
2014 Escobar G, Gentner B, Naldini L, Mazzieri R. Engineered tumor-infiltrating macrophages as gene delivery vehicles for interferon-α activates immunity and inhibits breast cancer progression. Oncoimmunology. 3: e28696. PMID 25097805 DOI: 10.4161/Onci.28696  0.346
2014 Friedli M, Turelli P, Kapopoulou A, Rauwel B, Castro-Díaz N, Rowe HM, Ecco G, Unzu C, Planet E, Lombardo A, Mangeat B, Wildhaber BE, Naldini L, Trono D. Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotency. Genome Research. 24: 1251-9. PMID 24879558 DOI: 10.1101/Gr.172809.114  0.392
2014 Genovese P, Schiroli G, Escobar G, Di Tomaso T, Firrito C, Calabria A, Moi D, Mazzieri R, Bonini C, Holmes MC, Gregory PD, van der Burg M, Gentner B, Montini E, Lombardo A, ... Naldini L, et al. Targeted genome editing in human repopulating haematopoietic stem cells. Nature. 510: 235-40. PMID 24870228 DOI: 10.1038/Nature13420  0.477
2014 Chiriaco M, Farinelli G, Capo V, Zonari E, Scaramuzza S, Di Matteo G, Sergi LS, Migliavacca M, Hernandez RJ, Bombelli F, Giorda E, Kajaste-Rudnitski A, Trono D, Grez M, Rossi P, ... ... Naldini L, et al. Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 1472-83. PMID 24869932 DOI: 10.1038/Mt.2014.87  0.466
2014 Rio P, Baños R, Lombardo A, Quintana-Bustamante O, Alvarez L, Garate Z, Genovese P, Almarza E, Valeri A, Díez B, Navarro S, Torres Y, Trujillo JP, Murillas R, Segovia JC, ... ... Naldini L, et al. Targeted gene therapy and cell reprogramming in Fanconi anemia. Embo Molecular Medicine. 6: 835-48. PMID 24859981 DOI: 10.15252/Emmm.201303374  0.449
2014 Lattanzi A, Salvagno C, Maderna C, Benedicenti F, Morena F, Kulik W, Naldini L, Montini E, Martino S, Gritti A. Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy. Human Molecular Genetics. 23: 3250-68. PMID 24463623 DOI: 10.1093/Hmg/Ddu034  0.474
2014 Cesana D, Ranzani M, Volpin M, Bartholomae C, Duros C, Artus A, Merella S, Benedicenti F, Sergi Sergi L, Sanvito F, Brombin C, Nonis A, Serio CD, Doglioni C, von Kalle C, ... ... Naldini L, et al. Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 774-85. PMID 24441399 DOI: 10.1038/Mt.2014.3  0.373
2014 Escobar G, Moi D, Ranghetti A, Ozkal-Baydin P, Squadrito ML, Kajaste-Rudnitski A, Bondanza A, Gentner B, De Palma M, Mazzieri R, Naldini L. Genetic engineering of hematopoiesis for targeted IFN-α delivery inhibits breast cancer progression. Science Translational Medicine. 6: 217ra3. PMID 24382895 DOI: 10.1126/Scitranslmed.3006353  0.434
2014 Biasco L, Scala S, Dionisio F, Calabria A, Ricci LB, Scaramuzza S, Baricordi C, Giannelli S, Neduva VX, Dow DJ, Pellin D, Vicard P, Serio CD, Montini E, Naldini L, et al. Comprehensive Clonal Mapping of Hematopoiesis in Vivo in Humans By Retroviral Vector Insertional Barcoding Blood. 124: 5-5. DOI: 10.1182/Blood.V124.21.5.5  0.471
2014 Mastaglio S, Genovese P, Magnani Z, Landoni E, Camisa B, Schiroli G, Provasi E, Lombardo A, Reik A, Cieri N, Ponzoni M, Ciceri F, Bordignon C, Holmes MC, Gregory PD, ... Naldini L, et al. NY-ESO-1 Single Edited T Cells to Treat Multiple Myeloma without Inducing GvHD Blood. 124: 308-308. DOI: 10.1182/Blood.V124.21.308.308  0.451
2014 Sauer AV, Visigalli I, Vezzoli M, Carriglio N, Ferro F, Delai S, Hernandez RJ, Mandelli G, Tiboni F, Rossi C, Palini A, Villa C, Sanvito F, Stradella E, Rodegher P, ... ... Naldini L, et al. Preclinical safety studies for gene therapy medicinal products in GLP test facility Toxicology Letters. 229. DOI: 10.1016/J.Toxlet.2014.06.538  0.345
2013 Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, ... ... Naldini L, et al. Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 175-184. PMID 28178602 DOI: 10.1038/mt.2012.23  0.378
2013 Passerini L, Rossi Mel E, Sartirana C, Fousteri G, Bondanza A, Naldini L, Roncarolo MG, Bacchetta R. CD4⁺ T cells from IPEX patients convert into functional and stable regulatory T cells by FOXP3 gene transfer. Science Translational Medicine. 5: 215ra174. PMID 24337481 DOI: 10.1126/Scitranslmed.3007320  0.411
2013 Annoni A, Cantore A, Della Valle P, Goudy K, Akbarpour M, Russo F, Bartolaccini S, D'Angelo A, Roncarolo MG, Naldini L. Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice. Embo Molecular Medicine. 5: 1684-97. PMID 24106222 DOI: 10.1002/Emmm.201302857  0.441
2013 Casucci M, Nicolis di Robilant B, Falcone L, Camisa B, Norelli M, Genovese P, Gentner B, Gullotta F, Ponzoni M, Bernardi M, Marcatti M, Saudemont A, Bordignon C, Savoldo B, Ciceri F, ... Naldini L, et al. CD44v6-targeted T cells mediate potent antitumor effects against acute myeloid leukemia and multiple myeloma. Blood. 122: 3461-72. PMID 24016461 DOI: 10.1182/Blood-2013-04-493361  0.452
2013 Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, ... ... Naldini L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science (New York, N.Y.). 341: 1233158. PMID 23845948 DOI: 10.1126/Science.1233158  0.478
2013 Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, Dionisio F, Calabria A, Giannelli S, Castiello MC, Bosticardo M, Evangelio C, Assanelli A, Casiraghi M, Di Nunzio S, ... ... Naldini L, et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (New York, N.Y.). 341: 1233151. PMID 23845947 DOI: 10.1126/Science.1233151  0.511
2013 Sonda N, Simonato F, Peranzoni E, Calì B, Bortoluzzi S, Bisognin A, Wang E, Marincola FM, Naldini L, Gentner B, Trautwein C, Sackett SD, Zanovello P, Molon B, Bronte V. miR-142-3p prevents macrophage differentiation during cancer-induced myelopoiesis. Immunity. 38: 1236-49. PMID 23809164 DOI: 10.1016/J.Immuni.2013.06.004  0.301
2013 Coluccio A, Miselli F, Lombardo A, Marconi A, Malagoli Tagliazucchi G, Gonçalves MA, Pincelli C, Maruggi G, Del Rio M, Naldini L, Larcher F, Mavilio F, Recchia A. Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1695-704. PMID 23760447 DOI: 10.1038/Mt.2013.143  0.477
2013 Patel AS, Smith A, Nucera S, Biziato D, Saha P, Attia RQ, Humphries J, Mattock K, Grover SP, Lyons OT, Guidotti LG, Siow R, Ivetic A, Egginton S, Waltham M, ... Naldini L, et al. TIE2-expressing monocytes/macrophages regulate revascularization of the ischemic limb. Embo Molecular Medicine. 5: 858-69. PMID 23653322 DOI: 10.1002/Emmm.201302752  0.301
2013 Amendola M, Giustacchini A, Gentner B, Naldini L. A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector). Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 934-46. PMID 23439497 DOI: 10.1038/Mt.2013.12  0.317
2013 Annoni A, Goudy K, Akbarpour M, Naldini L, Roncarolo MG. Immune responses in liver-directed lentiviral gene therapy. Translational Research : the Journal of Laboratory and Clinical Medicine. 161: 230-40. PMID 23360745 DOI: 10.1016/J.Trsl.2012.12.018  0.47
2013 Ranzani M, Cesana D, Bartholomae CC, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi LS, Merella S, Bulfone A, Doglioni C, von Kalle C, Kim YJ, Schmidt M, Tonon G, ... Naldini L, et al. Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer. Nature Methods. 10: 155-61. PMID 23314173 DOI: 10.1038/Nmeth.2331  0.381
2013 Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, ... ... Naldini L, et al. Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 175-84. PMID 22371846 DOI: 10.1038/Mt.2012.23  0.479
2013 Giustacchini A, Nucera S, Lechman ER, Fanciullo C, Boccalatte FE, Plati T, Ranghetti A, Vago L, Bernardi M, Ponzoni M, Dick JE, Naldini L, Gentner B. A Mechanistic Role For Mir-126, a Hematopoietic Stem Cell Microrna, In Acute Leukemias Blood. 122: 886-886. DOI: 10.1182/Blood.V122.21.886.886  0.309
2013 Robilant BNd, Casucci M, Falcone L, Camisa B, Gentner B, Naldini L, Bordignon C, Ciceri F, Bonini C, Bondanza A. CD44v6 Is Required For In Vivo Tumorigenesis Of Human AML and MM Cells: Role Of Microenvironmental Signals and Therapeutic Implications Blood. 122: 605-605. DOI: 10.1182/Blood.V122.21.605.605  0.426
2013 Mastaglio S, Genovese P, Magnani Z, Provasi E, Lombardo A, Reik A, Cieri N, Ciceri F, Bordignon C, Holmes MC, Gregory PD, Naldini L, Bonini C. TCR Gene Editing Achieved In a Single Round Of T Cell Activation Is Sufficient To Redirect T Cell Specificity and Prevent GvHD Blood. 122: 2898-2898. DOI: 10.1182/Blood.V122.21.2898.2898  0.442
2013 Buttiglieri S, Carlo-Stella C, Spatola T, Pulito R, Naldini L, Anichini A, Magni M, Giacomini A, Tarella C, Gianni AM. Potent In Vivo Anti-Tumor Activity Of Extracellular Vesicles Isolated From Genetically Engineered Primary Mesenchymal Stromal Cells Expressing The Trans-Membrane TNF-Related Apoptosis-Inducing Ligand (TRAIL) Blood. 122: 1658-1658. DOI: 10.1182/Blood.V122.21.1658.1658  0.398
2013 Lechman ER, Gentner B, van Galen P, Wai-Kwong Ng S, Eppert K, Marke R, Slush L, Kennedy JA, Mitchell A, Chen WC, Voisin V, Bader G, Minden MD, Lu J, Wang JC, ... Naldini L, et al. Mir-126 Governs Human Leukemia Stem Cell Quiescence and Chemotherapy Resistance Blood. 122: 1647-1647. DOI: 10.1182/Blood.V122.21.1647.1647  0.327
2013 Casucci M, Robilant BNd, Falcone L, Camisa B, Norelli M, Gentner B, Genovese P, Ponzoni M, Saudemont A, Bordignon C, Naldini L, Ciceri F, Dotti G, Bonini C, Bondanza A. Off-Tumor Target Expression Levels Do Not Predict CAR-T Cell Killing: A Foundation For The Safety Of CD44v6-Targeted T Cells Blood. 122: 142-142. DOI: 10.1182/Blood.V122.21.142.142  0.446
2013 Ranzani M, Cesana D, Bartholomä CC, Sanvito F, Riba M, Pala M, Benedicenti F, Gallina P, Annunziato S, Sergi LS, Merella S, Bulfone A, Doglioni C, Kalle Cv, Kim YJ, ... ... Naldini L, et al. Abstract 3169: Lentiviral vector-based insertional mutagenesis identifies new clinically relevant liver cancer genes. Cancer Research. 73: 3169-3169. DOI: 10.1158/1538-7445.Am2013-3169  0.381
2013 Ranzani M, Annunziato S, Benedicenti F, Gallina P, Naldini L, Montini E. Linear Amplification Mediated PCR for the retrieval of lentiviral vector integration sites from tumors induced by insertional mutagenesis. Protocol Exchange. DOI: 10.1038/Protex.2013.009  0.301
2013 Patel AS, Smith A, Nucera S, Biziato D, Saha P, Attia RQ, Humphries J, Grover SP, Mattock K, Lyons OT, Guidotti LG, Siow R, Ivetic A, Egginton S, Waltham M, ... Naldini L, et al. TIE2-expressing monocytes regulate revascularisation of the ischaemic limb The Lancet. 381. DOI: 10.1016/S0140-6736(13)60518-8  0.38
2012 Lechman ER, Gentner B, van Galen P, Giustacchini A, Saini M, Boccalatte FE, Hiramatsu H, Restuccia U, Bachi A, Voisin V, Bader GD, Dick JE, Naldini L. Attenuation of miR-126 activity expands HSC in vivo without exhaustion. Cell Stem Cell. 11: 799-811. PMID 23142521 DOI: 10.1016/J.Stem.2012.09.001  0.328
2012 Cantore A, Nair N, Della Valle P, Di Matteo M, Màtrai J, Sanvito F, Brombin C, Di Serio C, D'Angelo A, Chuah M, Naldini L, Vandendriessche T. Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. Blood. 120: 4517-20. PMID 23043073 DOI: 10.1182/Blood-2012-05-432591  0.434
2012 Capotondo A, Milazzo R, Politi LS, Quattrini A, Palini A, Plati T, Merella S, Nonis A, di Serio C, Montini E, Naldini L, Biffi A. Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proceedings of the National Academy of Sciences of the United States of America. 109: 15018-23. PMID 22923692 DOI: 10.1073/Pnas.1205858109  0.349
2012 Squadrito ML, Pucci F, Magri L, Moi D, Gilfillan GD, Ranghetti A, Casazza A, Mazzone M, Lyle R, Naldini L, De Palma M. miR-511-3p modulates genetic programs of tumor-associated macrophages. Cell Reports. 1: 141-54. PMID 22832163 DOI: 10.1016/J.Celrep.2011.12.005  0.318
2012 Torikai H, Reik A, Liu PQ, Zhou Y, Zhang L, Maiti S, Huls H, Miller JC, Kebriaei P, Rabinovitch B, Lee DA, Champlin RE, Bonini C, Naldini L, Rebar EJ, et al. A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR. Blood. 119: 5697-705. PMID 22535661 DOI: 10.1182/Blood-2012-01-405365  0.394
2012 Cesana D, Sgualdino J, Rudilosso L, Merella S, Naldini L, Montini E. Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. The Journal of Clinical Investigation. 122: 1667-76. PMID 22523064 DOI: 10.1172/Jci62189  0.433
2012 Provasi E, Genovese P, Lombardo A, Magnani Z, Liu PQ, Reik A, Chu V, Paschon DE, Zhang L, Kuball J, Camisa B, Bondanza A, Casorati G, Ponzoni M, Ciceri F, ... ... Naldini L, et al. Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer. Nature Medicine. 18: 807-15. PMID 22466705 DOI: 10.1038/Nm.2700  0.447
2012 Camnasio S, Delli Carri A, Lombardo A, Grad I, Mariotti C, Castucci A, Rozell B, Lo Riso P, Castiglioni V, Zuccato C, Rochon C, Takashima Y, Diaferia G, Biunno I, Gellera C, ... ... Naldini L, et al. The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity. Neurobiology of Disease. 46: 41-51. PMID 22405424 DOI: 10.1016/J.Nbd.2011.12.042  0.411
2012 Casucci M, Robilant BNd, Falcone L, Camisa B, Genovese P, Gentner B, Naldini L, Savoldo B, Ciceri F, Bordignon C, Dotti G, Bonini C, Bondanza A. Co-Expression of a Suicide Gene in CAR-Redirected T Cells Enables the Safe Targeting of CD44v6 for Leukemia and Myeloma Eradication Blood. 120: 949-949. DOI: 10.1182/Blood.V120.21.949.949  0.46
2012 Gentner B, Giustacchini A, Lechman E, Galen Pv, Boccalatte F, Saini M, Dick JE, Naldini L. Hematopoietic Stem Cell Expansion, without Exhaustion or Transformation, by Stable Microrna Antagonism in Vivo Blood. 120: 30-30. DOI: 10.1182/Blood.V120.21.30.30  0.382
2012 Ranzani M, Cesana D, Bartholomae CC, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi LS, Merella S, Bulfone A, Doglioni C, Kalle Cv, Kim YJ, Schmidt M, Tonon G, ... Naldini L, et al. Abstract 104: New liver cancer genes identified by lentiviral vector-based insertional mutagenesis in mice are associated to differential survival in hepatocellular carcinoma patients Cancer Research. 72: 104-104. DOI: 10.1158/1538-7445.Am2012-104  0.369
2012 Lechman ER, Gentner B, Galen Pv, Eppert K, Takenaka K, Minden M, Naldini L, Dick JE. Abstract 1013: Enriched miR-126 bioactivity marks the primitive compartment in human AML and regulates leukemia stem cell numbers Cancer Research. 72: 1013-1013. DOI: 10.1158/1538-7445.Am2012-1013  0.353
2011 Goudy KS, Annoni A, Naldini L, Roncarolo MG. Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy. Frontiers in Microbiology. 2: 221. PMID 22144977 DOI: 10.3389/Fmicb.2011.00221  0.46
2011 Passerini L, Olek S, Di Nunzio S, Barzaghi F, Hambleton S, Abinun M, Tommasini A, Vignola S, Cipolli M, Amendola M, Naldini L, Guidi L, Cecconi M, Roncarolo MG, Bacchetta R. Forkhead box protein 3 (FOXP3) mutations lead to increased TH17 cell numbers and regulatory T-cell instability. The Journal of Allergy and Clinical Immunology. 128: 1376-1379.e1. PMID 22000569 DOI: 10.1016/J.Jaci.2011.09.010  0.324
2011 Di Stefano B, Maffioletti SM, Gentner B, Ungaro F, Schira G, Naldini L, Broccoli V. A microRNA-based system for selecting and maintaining the pluripotent state in human induced pluripotent stem cells. Stem Cells (Dayton, Ohio). 29: 1684-95. PMID 21898693 DOI: 10.1002/Stem.726  0.404
2011 Lombardo A, Cesana D, Genovese P, Di Stefano B, Provasi E, Colombo DF, Neri M, Magnani Z, Cantore A, Lo Riso P, Damo M, Pello OM, Holmes MC, Gregory PD, Gritti A, ... ... Naldini L, et al. Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nature Methods. 8: 861-9. PMID 21857672 DOI: 10.1038/Nmeth.1674  0.503
2011 Gabriel R, Lombardo A, Arens A, Miller JC, Genovese P, Kaeppel C, Nowrouzi A, Bartholomae CC, Wang J, Friedman G, Holmes MC, Gregory PD, Glimm H, Schmidt M, Naldini L, et al. An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nature Biotechnology. 29: 816-23. PMID 21822255 DOI: 10.1038/Nbt.1948  0.311
2011 Neri M, Ricca A, di Girolamo I, Alcala'-Franco B, Cavazzin C, Orlacchio A, Martino S, Naldini L, Gritti A. Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy. Stem Cells (Dayton, Ohio). 29: 1559-71. PMID 21809420 DOI: 10.1002/Stem.701  0.455
2011 De Palma M, Naldini L. Angiopoietin-2 TIEs up macrophages in tumor angiogenesis. Clinical Cancer Research : An Official Journal of the American Association For Cancer Research. 17: 5226-32. PMID 21576085 DOI: 10.1158/1078-0432.Ccr-10-0171  0.32
2011 Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, ... ... Naldini L, et al. Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 1193-8. PMID 21540837 DOI: 10.1038/Mt.2011.78  0.484
2011 Bosticardo M, Draghici E, Schena F, Sauer AV, Fontana E, Castiello MC, Catucci M, Locci M, Naldini L, Aiuti A, Roncarolo MG, Poliani PL, Traggiai E, Villa A. Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome. The Journal of Allergy and Clinical Immunology. 127: 1376-84.e5. PMID 21531013 DOI: 10.1016/J.Jaci.2011.03.030  0.432
2011 Mátrai J, Cantore A, Bartholomae CC, Annoni A, Wang W, Acosta-Sanchez A, Samara-Kuko E, De Waele L, Ma L, Genovese P, Damo M, Arens A, Goudy K, Nichols TC, von Kalle C, ... ... Naldini L, et al. Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology (Baltimore, Md.). 53: 1696-707. PMID 21520180 DOI: 10.1002/Hep.24230  0.45
2011 Welford AF, Biziato D, Coffelt SB, Nucera S, Fisher M, Pucci F, Di Serio C, Naldini L, De Palma M, Tozer GM, Lewis CE. TIE2-expressing macrophages limit the therapeutic efficacy of the vascular-disrupting agent combretastatin A4 phosphate in mice. The Journal of Clinical Investigation. 121: 1969-73. PMID 21490397 DOI: 10.1172/Jci44562  0.309
2011 Mazzieri R, Pucci F, Moi D, Zonari E, Ranghetti A, Berti A, Politi LS, Gentner B, Brown JL, Naldini L, De Palma M. Targeting the ANG2/TIE2 axis inhibits tumor growth and metastasis by impairing angiogenesis and disabling rebounds of proangiogenic myeloid cells. Cancer Cell. 19: 512-26. PMID 21481792 DOI: 10.1016/J.Ccr.2011.02.005  0.364
2011 Naldini L. Ex vivo gene transfer and correction for cell-based therapies. Nature Reviews. Genetics. 12: 301-15. PMID 21445084 DOI: 10.1038/Nrg2985  0.477
2011 Biffi A, Bartolomae CC, Cesana D, Cartier N, Aubourg P, Ranzani M, Cesani M, Benedicenti F, Plati T, Rubagotti E, Merella S, Capotondo A, Sgualdino J, Zanetti G, von Kalle C, ... ... Naldini L, et al. Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood. 117: 5332-9. PMID 21403130 DOI: 10.1182/Blood-2010-09-306761  0.386
2011 Bartholomae CC, Arens A, Balaggan KS, Yáñez-Muñoz RJ, Montini E, Howe SJ, Paruzynski A, Korn B, Appelt JU, Macneil A, Cesana D, Abel U, Glimm H, Naldini L, Ali RR, et al. Lentiviral vector integration profiles differ in rodent postmitotic tissues. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 703-10. PMID 21364536 DOI: 10.1038/Mt.2011.19  0.473
2011 Pasi CE, Dereli-Öz A, Negrini S, Friedli M, Fragola G, Lombardo A, Van Houwe G, Naldini L, Casola S, Testa G, Trono D, Pelicci PG, Halazonetis TD. Genomic instability in induced stem cells. Cell Death and Differentiation. 18: 745-53. PMID 21311564 DOI: 10.1038/Cdd.2011.9  0.423
2011 Matsui H, Hegadorn C, Ozelo M, Burnett E, Tuttle A, Labelle A, McCray PB, Naldini L, Brown B, Hough C, Lillicrap D. A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 723-30. PMID 21285959 DOI: 10.1038/Mt.2010.290  0.419
2011 Casazza A, Fu X, Johansson I, Capparuccia L, Andersson F, Giustacchini A, Squadrito ML, Venneri MA, Mazzone M, Larsson E, Carmeliet P, De Palma M, Naldini L, Tamagnone L, Rolny C. Systemic and targeted delivery of semaphorin 3A inhibits tumor angiogenesis and progression in mouse tumor models. Arteriosclerosis, Thrombosis, and Vascular Biology. 31: 741-9. PMID 21205984 DOI: 10.1161/Atvbaha.110.211920  0.328
2011 Osborn MJ, McElmurry RT, Lees CJ, DeFeo AP, Chen ZY, Kay MA, Naldini L, Freeman G, Tolar J, Blazar BR. Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 450-60. PMID 21081900 DOI: 10.1038/Mt.2010.249  0.453
2011 Merten OW, Charrier S, Laroudie N, Fauchille S, Dugué C, Jenny C, Audit M, Zanta-Boussif MA, Chautard H, Radrizzani M, Vallanti G, Naldini L, Noguiez-Hellin P, Galy A. Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. Human Gene Therapy. 22: 343-56. PMID 21043787 DOI: 10.1089/Hum.2010.060  0.368
2011 Rossetti M, Gregori S, Hauben E, Brown BD, Sergi LS, Naldini L, Roncarolo MG. HIV-1-derived lentiviral vectors directly activate plasmacytoid dendritic cells, which in turn induce the maturation of myeloid dendritic cells. Human Gene Therapy. 22: 177-88. PMID 20825284 DOI: 10.1089/Hum.2010.085  0.382
2011 Montini E, Sgualdino J, Cesana D, Benedicenti F, Merella S, Leo S, Zanetti G, Naldini L. Assessing the impact of lentiviral vector integration on splicing of cellular genes at the genome-wide level Retrovirology. 8: 1-2. DOI: 10.1186/1742-4690-8-S2-O15  0.348
2011 Provasi E, Genovese P, Lombardo A, Magnani Z, Liu P, Reik A, Chu V, Paschon DE, Zhang L, Kuball J, Camisa B, Bondanza A, Casorati G, Ponzoni M, Ciceri F, ... ... Naldini L, et al. TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD Blood. 118: 667-667. DOI: 10.1182/Blood.V118.21.667.667  0.47
2011 Casucci M, Falcone L, Robilant BNd, Camisa B, Genovese P, Gentner B, Naldini L, Savoldo B, Ciceri F, Bordignon C, Dotti G, Bonini C, Bondanza A. Dual Transgenesis of T Cells with a Novel CD44v6-Specific Chimeric Antigen Receptor and a Suicide Gene for Safe and Effective Targeting of Chemoresistance in Hematopoietic Tumors Blood. 118: 3125-3125. DOI: 10.1182/Blood.V118.21.3125.3125  0.476
2011 Ranzani M, Cesana D, Bartholomae C, Sanvito F, Pala M, Benedicenti F, Sergi LS, Bulfone A, Doglioni C, Kalle Cv, Schmidt M, Tonon G, Naldini L, Montini E. Abstract 4982: Identification of new human liver cancer genes by a novel lentiviral vector-based insertional mutagenesis approach in three mouse models of hepatocarcinogenesis Cancer Research. 71: 4982-4982. DOI: 10.1158/1538-7445.Am2011-4982  0.363
2010 Gentner B, Visigalli I, Hiramatsu H, Lechman E, Ungari S, Giustacchini A, Schira G, Amendola M, Quattrini A, Martino S, Orlacchio A, Dick JE, Biffi A, Naldini L. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Science Translational Medicine. 2: 58ra84. PMID 21084719 DOI: 10.1126/Scitranslmed.3001522  0.483
2010 Visigalli I, Delai S, Politi LS, Di Domenico C, Cerri F, Mrak E, D'Isa R, Ungaro D, Stok M, Sanvito F, Mariani E, Staszewsky L, Godi C, Russo I, Cecere F, ... ... Naldini L, et al. Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood. 116: 5130-9. PMID 20847202 DOI: 10.1182/Blood-2010-04-278234  0.433
2010 Sachdeva R, Jönsson ME, Nelander J, Kirkeby A, Guibentif C, Gentner B, Naldini L, Björklund A, Parmar M, Jakobsson J. Tracking differentiating neural progenitors in pluripotent cultures using microRNA-regulated lentiviral vectors. Proceedings of the National Academy of Sciences of the United States of America. 107: 11602-7. PMID 20534548 DOI: 10.1073/Pnas.1006568107  0.437
2010 Visigalli I, Ungari S, Martino S, Park H, Cesani M, Gentner B, Sergi Sergi L, Orlacchio A, Naldini L, Biffi A. The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche. Blood. 116: 1857-66. PMID 20511539 DOI: 10.1182/Blood-2009-12-256461  0.381
2010 Lattanzi A, Neri M, Maderna C, di Girolamo I, Martino S, Orlacchio A, Amendola M, Naldini L, Gritti A. Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models. Human Molecular Genetics. 19: 2208-27. PMID 20203170 DOI: 10.1093/Hmg/Ddq099  0.405
2010 Coffelt SB, Lewis CE, Naldini L, Brown JM, Ferrara N, De Palma M. Elusive identities and overlapping phenotypes of proangiogenic myeloid cells in tumors. The American Journal of Pathology. 176: 1564-76. PMID 20167863 DOI: 10.2353/Ajpath.2010.090786  0.369
2010 Andreu P, Johansson M, Affara NI, Pucci F, Tan T, Junankar S, Korets L, Lam J, Tawfik D, DeNardo DG, Naldini L, de Visser KE, De Palma M, Coussens LM. FcRgamma activation regulates inflammation-associated squamous carcinogenesis. Cancer Cell. 17: 121-34. PMID 20138013 DOI: 10.1016/J.Ccr.2009.12.019  0.318
2010 Lechman E, Gentner B, Hiramatsu H, Hope KJ, Takenaka K, Eppert K, Minden MD, Naldini L, Dick JE. Enriched MicroRNA-126 Bioactivity Marks the Primitive Compartment In AML and Regulates LSC Numbers Blood. 116: 94-94. DOI: 10.1182/Blood.V116.21.94.94  0.354
2010 Provasi E, Genovese P, Lombardo A, Magnani Z, Reik A, Liu P, Chu V, Kuball J, Bondanza A, Casorati G, Ciceri F, Bordignon C, Greenberg PD, Holmes MC, Gregory PD, ... Naldini L, et al. Editing Human Lymphocyte Specificity for Safe and Effective Adoptive Immunotherapy of Leukemia. Blood. 116: 3764-3764. DOI: 10.1182/Blood.V116.21.3764.3764  0.462
2010 Gentner B, Giustacchini A, Boccalatte F, Schira G, Saini M, Lechman E, Hiramatsu H, Dick JE, Naldini L. Identification and Function of Hematopoietic Stem and Progenitor Cell Specific Micrornas. Blood. 116: 2631-2631. DOI: 10.1182/Blood.V116.21.2631.2631  0.334
2010 Genovese P, Provasi E, Magnani Z, Lombardo A, Pello OM, Kuball J, Bondanza A, Gregory PD, Bordignon C, Holmes MC, Greenberg PD, Naldini L, Bonini C. Abstract 2937: Editing central memory T lymphocyte specificity for safe and effective adoptive immunotherapy of leukemia Cancer Research. 70: 2937-2937. DOI: 10.1158/1538-7445.Am10-2937  0.488
2009 Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, ... Naldini L, et al. Corrigendum to "Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models". Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1300. PMID 28178476 DOI: 10.1038/Mt.2009.61  0.334
2009 Bobisse S, Rondina M, Merlo A, Tisato V, Mandruzzato S, Amendola M, Naldini L, Willemsen RA, Debets R, Zanovello P, Rosato A. Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors. Cancer Research. 69: 9385-94. PMID 19996290 DOI: 10.1158/0008-5472.Can-09-0494  0.465
2009 Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, ... ... Naldini L, et al. Comprehensive genomic access to vector integration in clinical gene therapy. Nature Medicine. 15: 1431-6. PMID 19966782 DOI: 10.1038/Nm.2057  0.384
2009 Naldini L. Medicine. A comeback for gene therapy. Science (New York, N.Y.). 326: 805-6. PMID 19892968 DOI: 10.1126/Science.1181937  0.458
2009 Annoni A, Brown BD, Cantore A, Sergi LS, Naldini L, Roncarolo MG. In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. Blood. 114: 5152-61. PMID 19794140 DOI: 10.1182/Blood-2009-04-214569  0.378
2009 Cassani B, Montini E, Maruggi G, Ambrosi A, Mirolo M, Selleri S, Biral E, Frugnoli I, Hernandez-Trujillo V, Di Serio C, Roncarolo MG, Naldini L, Mavilio F, Aiuti A. Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood. 114: 3546-56. PMID 19652199 DOI: 10.1182/Blood-2009-02-202085  0.464
2009 Denard J, Rundwasser S, Laroudie N, Gonnet F, Naldini L, Radrizzani M, Galy A, Merten OW, Danos O, Svinartchouk F. Quantitative proteomic analysis of lentiviral vectors using 2-DE. Proteomics. 9: 3666-76. PMID 19639585 DOI: 10.1002/Pmic.200800747  0.317
2009 Plati T, Visigalli I, Capotondo A, Buono M, Naldini L, Cosma MP, Biffi A. Development and maturation of invariant NKT cells in the presence of lysosomal engulfment. European Journal of Immunology. 39: 2748-54. PMID 19637231 DOI: 10.1002/Eji.200939639  0.35
2009 Brown BD, Naldini L. Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nature Reviews. Genetics. 10: 578-85. PMID 19609263 DOI: 10.1038/Nrg2628  0.358
2009 Cesani M, Capotondo A, Plati T, Sergi LS, Fumagalli F, Roncarolo MG, Naldini L, Comi G, Sessa M, Biffi A. Characterization of new arylsulfatase A gene mutations reinforces genotype-phenotype correlation in metachromatic leukodystrophy. Human Mutation. 30: E936-45. PMID 19606494 DOI: 10.1002/Humu.21093  0.302
2009 Pucci F, Venneri MA, Biziato D, Nonis A, Moi D, Sica A, Di Serio C, Naldini L, De Palma M. A distinguishing gene signature shared by tumor-infiltrating Tie2-expressing monocytes, blood "resident" monocytes, and embryonic macrophages suggests common functions and developmental relationships. Blood. 114: 901-14. PMID 19383967 DOI: 10.1182/Blood-2009-01-200931  0.395
2009 De Palma M, Naldini L. Tie2-expressing monocytes (TEMs): novel targets and vehicles of anticancer therapy? Biochimica Et Biophysica Acta. 1796: 5-10. PMID 19362584 DOI: 10.1016/J.Bbcan.2009.04.001  0.382
2009 Visigalli I, Moresco RM, Belloli S, Politi LS, Gritti A, Ungaro D, Matarrese M, Turolla E, Falini A, Scotti G, Naldini L, Fazio F, Biffi A. Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195. Neurobiology of Disease. 34: 51-62. PMID 19320046 DOI: 10.1016/J.Nbd.2008.12.019  0.37
2009 Montini E, Cesana D, Schmidt M, Sanvito F, Bartholomae CC, Ranzani M, Benedicenti F, Sergi LS, Ambrosi A, Ponzoni M, Doglioni C, Di Serio C, von Kalle C, Naldini L. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. The Journal of Clinical Investigation. 119: 964-75. PMID 19307726 DOI: 10.1172/Jci37630  0.47
2009 Amendola M, Passerini L, Pucci F, Gentner B, Bacchetta R, Naldini L. Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1039-52. PMID 19293777 DOI: 10.1038/Mt.2009.48  0.317
2009 Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, ... Naldini L, et al. Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1073-82. PMID 19259069 DOI: 10.1038/Mt.2009.31  0.474
2009 Carlén M, Meletis K, Göritz C, Darsalia V, Evergren E, Tanigaki K, Amendola M, Barnabé-Heider F, Yeung MS, Naldini L, Honjo T, Kokaia Z, Shupliakov O, Cassidy RM, Lindvall O, et al. Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke. Nature Neuroscience. 12: 259-67. PMID 19234458 DOI: 10.1038/Nn.2268  0.334
2009 Santoni de Sio F, Naldini L. Short-term culture of human CD34+ cells for lentiviral gene transfer. Methods in Molecular Biology (Clifton, N.J.). 506: 59-70. PMID 19110619 DOI: 10.1007/978-1-59745-409-4_5  0.506
2009 Gentner B, Schira G, Giustacchini A, Amendola M, Brown BD, Ponzoni M, Naldini L. Stable knockdown of microRNA in vivo by lentiviral vectors. Nature Methods. 6: 63-6. PMID 19043411 DOI: 10.1038/Nmeth.1277  0.33
2009 Provasi E, Genovese P, Magnani Z, Pello OM, Kuball J, Lombardo A, Bondanza A, Gregory PD, Bordignon C, Holmes MC, Greenberg PD, Naldini L, Bonini C. From TCR Gene Transfer to TCR Gene Editing of Central Memory T Lymphocytes for Immunotherapy of Leukemia. Blood. 114: 374-374. DOI: 10.1182/Blood.V114.22.374.374  0.477
2008 De Palma M, Mazzieri R, Politi LS, Pucci F, Zonari E, Sitia G, Mazzoleni S, Moi D, Venneri MA, Indraccolo S, Falini A, Guidotti LG, Galli R, Naldini L. Tumor-targeted interferon-alpha delivery by Tie2-expressing monocytes inhibits tumor growth and metastasis. Cancer Cell. 14: 299-311. PMID 18835032 DOI: 10.1016/J.Ccr.2008.09.004  0.37
2008 Wilson JM, Gansbacher B, Berns KI, Bosch F, Kay MA, Naldini L, Wei YQ. Good news on the clinical gene transfer front. Human Gene Therapy. 19: 429-30. PMID 18507511 DOI: 10.1089/Hum.2008.0519  0.343
2008 Santoni de Sio FR, Gritti A, Cascio P, Neri M, Sampaolesi M, Galli C, Luban J, Naldini L. Lentiviral vector gene transfer is limited by the proteasome at postentry steps in various types of stem cells. Stem Cells (Dayton, Ohio). 26: 2142-52. PMID 18483423 DOI: 10.1634/Stemcells.2007-0705  0.478
2008 Allan SE, Alstad AN, Merindol N, Crellin NK, Amendola M, Bacchetta R, Naldini L, Roncarolo MG, Soudeyns H, Levings MK. Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 194-202. PMID 17984976 DOI: 10.1038/Sj.Mt.6300341  0.434
2008 Lechman ER, Hiramatsu H, Gentner B, Hope K, Takenaka K, Eppert K, Minden MD, Naldini L, Dick JE. High Levels of MicroRNA-126 Bioactivity Specify the LSC Compartment in AML Blood. 112: 510-510. DOI: 10.1182/Blood.V112.11.510.510  0.35
2008 Provasi E, Pello OM, Magnani Z, Kuball J, Lombardo A, Bondanza A, Gregory PD, Bordignon C, Holmes MC, Greenberg PD, Naldini L, Bonini C. T Cell Receptor Gene Transfer into Naive and Central Memory Lymphocytes by Lentiviral Vectors for a Safe and Effective Adoptive Immune Therapy of Leukemia Blood. 112: 3529-3529. DOI: 10.1182/Blood.V112.11.3529.3529  0.456
2008 Visigalli I, Cesani M, Neri M, Ungari S, Tononi L, Marchesini S, Grez M, Gritti A, Naldini L, Biffi A. 103. Development of an effective and safe hematopoietic stem cell gene therapy for globoid cell leukodystrophy: The unexpected issue of GALC toxicity Molecular Genetics and Metabolism. 93: 41. DOI: 10.1016/J.Ymgme.2007.10.115  0.464
2008 Biffi A, Capotondo A, Cesani M, Montini E, Plati T, Benedicenti F, Tononi L, Marchesini S, Sessa M, Bordignon C, Roncarolo MG, Naldini L. 9. Hematopoietic stem cell based gene therapy for the treatment of metachromatic leukodystrophy: Towards clinical testing Molecular Genetics and Metabolism. 93: 16. DOI: 10.1016/J.Ymgme.2007.10.021  0.425
2008 Roncarolo MGG, Bosticardo M, Marangoni F, Scaramuzza S, Trifari S, Galy A, Dupré L, Naldini L, Aiuti A, Villa A. Evidence for efficacy and safety of lentiviral mediated gene transfer in Wiskott–Aldrich syndrome Blood Cells Molecules and Diseases. 40: 282. DOI: 10.1016/J.Bcmd.2007.10.072  0.328
2008 Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Sergi LS, Benedicenti F, Bartholomae C, Serio CD, Doglioni C, Kalle Cv, Naldini L. Modeling the genotoxicity of viral vector integration in a tumor prone hematopoietic stem cell transplantation model Blood Cells Molecules and Diseases. 40: 276-277. DOI: 10.1016/J.Bcmd.2007.10.061  0.373
2008 Gentner B, Colleoni S, Brown BD, Gritti A, Galli C, Naldini L. Exploiting microRNA expression profiles for lineage- and differentiation state-specific transgene expression in hematopoietic, neural and embryonic stem cells Blood Cells Molecules and Diseases. 40: 267. DOI: 10.1016/J.Bcmd.2007.10.042  0.361
2008 Bosticardo M, Marangoni F, Locci M, Draghici E, Scaramuzza S, Panaroni C, Galy A, Naldini L, Aiuti A, Dupré L, Roncarolo MG, Anna. Long-term efficacy and safety of hematopoietic stem cell gene therapy mediated by lentiviral vectors in the murine model of Wiskott-Aldrich syndrome Blood Cells Molecules and Diseases. 40: 256-257. DOI: 10.1016/J.Bcmd.2007.10.023  0.429
2007 Brown BD, Gentner B, Cantore A, Colleoni S, Amendola M, Zingale A, Baccarini A, Lazzari G, Galli C, Naldini L. Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nature Biotechnology. 25: 1457-67. PMID 18026085 DOI: 10.1038/Nbt1372  0.35
2007 De Palma M, Murdoch C, Venneri MA, Naldini L, Lewis CE. Tie2-expressing monocytes: regulation of tumor angiogenesis and therapeutic implications. Trends in Immunology. 28: 519-24. PMID 17981504 DOI: 10.1016/J.It.2007.09.004  0.328
2007 Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee YL, Kim KA, Ando D, Urnov FD, Galli C, Gregory PD, Holmes MC, Naldini L. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nature Biotechnology. 25: 1298-306. PMID 17965707 DOI: 10.1016/J.Bcmd.2007.10.064  0.492
2007 Capotondo A, Cesani M, Pepe S, Fasano S, Gregori S, Tononi L, Venneri MA, Brambilla R, Quattrini A, Ballabio A, Cosma MP, Naldini L, Biffi A. Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy. Human Gene Therapy. 18: 821-36. PMID 17845130 DOI: 10.1089/Hum.2007.048  0.49
2007 Brown BD, Cantore A, Annoni A, Sergi LS, Lombardo A, Della Valle P, D'Angelo A, Naldini L. A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice Blood. 110: 4144-4152. PMID 17726165 DOI: 10.1182/Blood-2007-03-078493  0.398
2007 Biffi A, Naldini L. Novel candidate disease for gene therapy: Metachromatic leukodystrophy Expert Opinion On Biological Therapy. 7: 1193-1205. PMID 17696818 DOI: 10.1517/14712598.7.8.1193  0.429
2007 Annoni A, Battaglia M, Follenzi A, Lombardo A, Sergi-Sergi L, Naldini L, Roncarolo MG. The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4 +CD25+ regulatory T cells Blood. 110: 1788-1796. PMID 17495135 DOI: 10.1182/Blood-2006-11-059873  0.406
2007 Venneri MA, De Palma M, Ponzoni M, Pucci F, Scielzo C, Zonari E, Mazzieri R, Doglioni C, Naldini L. Identification of proangiogenic TIE2-expressing monocytes (TEMs) in human peripheral blood and cancer. Blood. 109: 5276-85. PMID 17327411 DOI: 10.1182/Blood-2006-10-053504  0.356
2007 Fraldi A, Biffi A, Lombardi A, Visigalli I, Pepe S, Settembre C, Nusco E, Auricchio A, Naldini L, Ballabio A, Cosma MP. SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies. The Biochemical Journal. 403: 305-12. PMID 17206939 DOI: 10.1042/Bj20061783  0.427
2007 Brown BD, Sitia G, Annoni A, Hauben E, Sergi LS, Zingale A, Roncarolo MG, Guidotti LG, Naldini L. In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance Blood. 109: 2797-2805. PMID 17170119 DOI: 10.1182/Blood-2006-10-049312  0.484
2007 Matsui H, Ozelo M, Hegadorn C, Labelle A, Burnett E, Tuttle A, Brown B, Naldini L, Hough C, Lillicrap D. Long-Term Phenotypic Correction of Hemophilia A Mice Following Intravenous Injection of miRNA-Regulated Lentiviral Vectors. Blood. 110: 2587-2587. DOI: 10.1182/Blood.V110.11.2587.2587  0.432
2007 Naldini L, Palma MD, Venneri MA, Pucci F, Galli R, Politi SL, Sitia G. 5 INVITED Role of haematopoietic cells in tumour angiogenesis: from discovery to targeted cancer gene therapy Ejc Supplements. 5: 4-5. DOI: 10.1016/S1359-6349(07)70119-2  0.404
2006 Loew R, Vigna E, Lindemann D, Naldini L, Bujard H. Retroviral vectors containing Tet-controlled bidirectional transcription units for simultaneous regulation of two gene activities. Journal of Molecular and Genetic Medicine. 2: 107-118. PMID 19565004 DOI: 10.4172/1747-0862.1000021  0.461
2006 Biffi A, Capotondo A, Fasano S, del Carro U, Marchesini S, Azuma H, Malaguti MC, Amadio S, Brambilla R, Grompe M, Bordignon C, Quattrini A, Naldini L. Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. The Journal of Clinical Investigation. 116: 3070-82. PMID 17080200 DOI: 10.1172/Jci28873  0.442
2006 Di Domenico C, Di Napoli D, Gonzalez Y Reyero E, Lombardo A, Naldini L, Di Natale P. Limited transgene immune response and long-term expression of human α-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy Human Gene Therapy. 17: 1112-1121. PMID 17044753 DOI: 10.1089/Hum.2006.17.1112  0.393
2006 De Palma M, Naldini L. Role of haematopoietic cells and endothelial progenitors in tumour angiogenesis Biochimica Et Biophysica Acta - Reviews On Cancer. 1766: 159-166. PMID 16857321 DOI: 10.1016/J.Bbcan.2006.06.003  0.328
2006 Mortellaro A, Hernandez RJ, Guerrini MM, Carlucci F, Tabucchi A, Ponzoni M, Sanvito F, Doglioni C, Di Serio C, Biasco L, Follenzi A, Naldini L, Bordignon C, Roncarolo MG, Aiuti A. Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects Blood. 108: 2979-2988. PMID 16835374 DOI: 10.1182/Blood-2006-05-023507  0.462
2006 Dolcetta D, Perani L, Givogri MI, Galbiati F, Amadio S, Del Carro U, Finocchiaro G, Fanzani A, Marchesini S, Naldini L, Roncarolo MG, Bongarzone E. Design and optimization of lentiviral vectors for transfer of GALC expression in Twitcher brain. The Journal of Gene Medicine. 8: 962-71. PMID 16732552 DOI: 10.1002/Jgm.924  0.447
2006 Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Bartholomae C, Sergi LS, Benedicenti F, Ambrosi A, Di Serio C, Doglioni C, Von Kalle C, Naldini L. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration Nature Biotechnology. 24: 687-696. PMID 16732270 DOI: 10.1038/Nbt1216  0.501
2006 Patanè S, Avnet S, Coltella N, Costa B, Sponza S, Olivero M, Vigna E, Naldini L, Baldini N, Ferracini R, Corso S, Giordano S, Comoglio PM, Di Renzo MF. MET overexpression turns human primary osteoblasts into osteosarcomas. Cancer Research. 66: 4750-7. PMID 16651428 DOI: 10.1158/0008-5472.Can-05-4422  0.377
2006 Brown BD, Venneri MA, Zingale A, Sergi LS, Naldini L. Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer Nature Medicine. 12: 585-591. PMID 16633348 DOI: 10.1038/Nm1398  0.382
2006 Naldini L. Inserting optimism into gene therapy Nature Medicine. 12: 386-388. PMID 16598281 DOI: 10.1038/Nm0406-386  0.384
2006 Dupré L, Marangoni F, Scaramuzza S, Trifari S, Hernandez RJ, Aiuti A, Naldini L, Roncarolo MG. Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation Human Gene Therapy. 17: 303-313. PMID 16544979 DOI: 10.1089/Hum.2006.17.303  0.479
2006 Santoni De Sio FR, Cascio P, Zingale A, Gasparini M, Naldini L. Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction Blood. 107: 4257-4265. PMID 16469870 DOI: 10.1182/Blood-2005-10-4047  0.5
2006 Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Sergi LS, Benedicenti F, Bartholomae C, Serio CD, Doglioni C, Kalle Cv, Naldini L. Modeling the Genotoxicity of Viral Vector Integration in a Tumor Prone Hematopoietic Stem Cell Transplantation Model. Blood. 108: 451-451. DOI: 10.1182/Blood.V108.11.451.451  0.458
2006 Scaramuzza S, Trifari S, Marangoni F, Martino S, Metin A, Galy A, Naldini L, Aiuti A, Villa A, Dupré L, Roncarolo MG. Evidence for Efficacy and Safety of Lentiviral Mediated Gene Transfer in T Cells and CD34+ Cells from Wiskott-Aldrich Syndrome Patients. Blood. 108: 3279-3279. DOI: 10.1182/Blood.V108.11.3279.3279  0.488
2006 Biffi A, Capotondo A, Fasano S, del Carro U, Marchesini S, Azuma H, Brambilla R, Grompe M, Bordignon C, Quattrini A, Naldini L. 891. Correction of Established Neurologic Disease and Evidences of In Vivo Cross Correction in the Mouse Model of Metachromatic Leukodystrophy Molecular Therapy. 13: S343. DOI: 10.1016/J.Ymthe.2006.08.980  0.44
2006 Visigalli I, Carro Ud, Politi L, Quattrini A, Luzi P, Wenger D, Naldini L, Biffi A. 888. Characterization of New Murine Models of Globoid Cell Leukodystrophy: Relevance for Gene Therapy Applications and Studies on Disease Pathogenesis Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.977  0.376
2006 Capotondo A, Venneri MA, Cosma MP, Pallavicini I, Ballabio A, Naldini L, Biffi A. 864. Characterization of Transgenic Mice Over_ Expressing Arylsulfatase A: Implications for Gene Therapy and Sulfatases Activation Mechanisms Molecular Therapy. 13: S332-S333. DOI: 10.1016/J.Ymthe.2006.08.950  0.419
2006 Lombardo A, Beausejour C, Urnov FD, Miller JC, Aiuti A, Gregory PD, Holmes MC, Naldini L. 738. Towards Gene Correction of X-Linked SCID Using Engineered Zinc Finger Nucleases and Integrase Defective Lentiviral Delivery Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.819  0.447
2006 Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Sergi LS, Benedicenti F, Bartholomae C, Serio CD, Doglioni C, Kalle Cv, Naldini L. 731. Hematopoietic Stem Cell Gene Transfer and Integration Site Analysis in Tumor-Prone Mice Uncovers Low Genotoxicity of Lentiviral Vector Integration Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.812  0.438
2006 Copreni E, Palmieri L, Carrabino S, Castellani S, Naldini L, Conese M. 694. Involvement of Glycosaminoglycans in VSV-G Pseudotyped Lentiviral Vector Mediated Gene Transfer into Airway Epithelial Cells Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.772  0.446
2006 Amendola M, Politi LS, Cadioli M, Galli R, Binda E, Falini A, Levi S, Scotti G, Biffi A, Naldini L. 442. LV Expressing MR Reporter Genes Allows In Vivo Monitoring of Stem Cell Gene Therapy Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.509  0.494
2006 Biffi A, Visigalli I, Cesani M, Capotondo A, Sio FRSd, Pallavicini I, Zingale A, Naldini L. 408. Safety of Lysosomal Enzymes Over-Expression in HSC for Gene Therapy of Storage Disorders Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.471  0.489
2006 Urnov FD, Lombardo A, Rock J, Moehle E, Miller JC, Lee Y, Gregory PD, Naldini L, Holmes MC. 1003. Targeted Site-Specific Integration in Human Cells Using Designed Zinc Finger Nucleases Molecular Therapy. 13: S386-S387. DOI: 10.1016/J.Ymthe.2006.08.1097  0.384
2006 Annoni A, Brown B, Battaglia M, Lombardo A, Naldini L, Roncarolo MG. 981. Prevention of Transgene Expression in Antigen Presenting Cells Correlate with Modulated Immune Response after In Vivo Gene Transfer Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.1074  0.447
2006 Brown BD, Hauben E, Sitia G, Zingale A, Sergi LS, Guidotti L, Roncarolo MG, Naldini L. 79. Systemic Administration of Lentiviral Vectors Triggers Innate Host Responses Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.096  0.323
2006 Marangoni F, Dupré L, Scaramuzza S, Panaroni C, Trifari S, Hernández RJ, Thrasher AJ, Galy A, Aiuti A, Naldini L, Roncarolo MG. 65. Long-Term Effects of Hematopoietic Stem Cell Gene Therapy in the Murine Model of Wiskott-Aldrich Syndrome: Persistence of Functional Correction of T Cells and Lack of Malignant Trasformation Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.081  0.475
2006 DePalma M, Galli R, Venneri MA, Politi LS, Pucci F, Sergi LS, Binda E, Hauben E, Naldini L. 57. Targeted Gene Delivery of Alpha-Interferon by Genetically Modified Hematopoietic Cells Inhibits Glioma Vascularization and Growth without Systemic Toxicity Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.072  0.405
2006 Allan S, Passerini L, Bacchetta R, Amendola M, Naldini L, Roncarolo MG, Levings M. Su.98. Defining a Role for FOXP3 in Human Cd4+ T-Cells Clinical Immunology. 119. DOI: 10.1016/J.Clim.2006.04.525  0.317
2005 Biffi A, Naldini L. Gene therapy of storage disorders by retroviral and lentiviral vectors Human Gene Therapy. 16: 1133-1142. PMID 16218774 DOI: 10.1089/Hum.2005.16.1133  0.385
2005 De Palma M, Venneri MA, Galli R, Sergi LS, Politi LS, Sampaolesi M, Naldini L. Tie2 identifies a hematopoietic lineage of proangiogenic monocytes required for tumor vessel formation and a mesenchymal population of pericyte progenitors Cancer Cell. 8: 211-226. PMID 16169466 DOI: 10.1016/J.Ccr.2005.08.002  0.369
2005 Abordo-Adesida E, Follenzi A, Barcia C, Sciascia S, Castro MG, Naldini L, Lowenstein PR. Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses. Human Gene Therapy. 16: 741-51. PMID 15960605 DOI: 10.1089/Hum.2005.16.741  0.362
2005 Cignetti A, Vallario A, Follenzi A, Circosta P, Capaldi A, Gottardi D, Naldini L, Caligaris-Cappio F. Lentiviral transduction of primary myeloma cells with CD80 and CD154 generates antimyeloma effector T cells. Human Gene Therapy. 16: 445-56. PMID 15871676 DOI: 10.1089/Hum.2005.16.445  0.372
2005 Vigna E, Amendola M, Benedicenti F, Simmons AD, Follenzi A, Naldini L. Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector Molecular Therapy. 11: 763-775. PMID 15851015 DOI: 10.1016/J.Ymthe.2004.11.017  0.425
2005 Boccaccio C, Sabatino G, Medico E, Girolami F, Follenzi A, Reato G, Sottile A, Naldini L, Comoglio PM. The MET oncogene drives a genetic programme linking cancer to haemostasis Nature. 434: 396-400. PMID 15772665 DOI: 10.1038/Nature03357  0.337
2005 Taulli R, Accornero P, Follenzi A, Mangano T, Morotti A, Scuoppo C, Forni PE, Bersani F, Crepaldi T, Chiarle R, Naldini L, Ponzetto C. RNAi technology and lentiviral delivery as a powerful tool to suppress Tpr-Met-mediated tumorigenesis Cancer Gene Therapy. 12: 456-463. PMID 15719029 DOI: 10.1038/Sj.Cgt.7700815  0.314
2005 Di Domenico C, Villani GRD, Di Napoli D, Gonzalez Y Reyero E, Lombardo A, Naldini L, Di Natale P. Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector Human Gene Therapy. 16: 81-90. PMID 15703491 DOI: 10.1089/Hum.2005.16.81  0.374
2005 Di Natale P, Di Domenico C, Gargiulo N, Castaldo S, Gonzalez Y. Reyero E, Mithbaokar P, De Felice M, Follenzi A, Naldini L, Villani GRD. Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector Biochemical Journal. 388: 639-646. PMID 15649123 DOI: 10.1042/Bj20041702  0.431
2005 Amendola M, Venneri MA, Biffi A, Vigna E, Naldini L. Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters Nature Biotechnology. 23: 108-116. PMID 15619618 DOI: 10.1038/Nbt1049  0.459
2005 De Palma M, Montini E, Santoni De Sio FR, Benedicenti F, Gentile A, Medico E, Naldini L. Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells Blood. 105: 2307-2315. PMID 15542582 DOI: 10.1182/Blood-2004-03-0798  0.397
2005 Cohen-Haguenauer OYD, Bosch F, Carrondo M, Cichutek K, Deglon N, Demeneix B, Dickson G, Garhton G, Gansbacher B, Melani C, Naldini L, Rosenthal F, Rusconi S, Yla-Herttuala S, Zwierzina H. 1070. Easy Search on Clinical Gene Transfer: The Euregenethy (EC-DG Research FP5-TN) Data Base on Pre-Clinical & Clinical Gene Transfer & Therapy Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.617  0.375
2005 Sio FSd, Naldini L. 1038. Lentiviral Gene Transfer into HSC Is Enhanced by Early-Acting Cytokines without Impairing Stem Cell Properties and Involves Cellular Responses Distinct from Cell Cycle Control Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.585  0.506
2005 Biffi A, Visigalli I, Politi LS, Quattrini A, Carro UD, Wenger D, Bordignon C, Naldini L. 972. HSC-Based Ex Vivo Gene Therapy Prolongs Survival and Improves the Phenotype of Globoid Cell Leukodystrophy Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.515  0.453
2005 Annoni A, Battaglia M, Follenzi A, Lombardo A, Naldini L, Roncarolo MG. 468. Cellular Therapy with Transgene Expressing APC Activates CD4+CD25+ Regulatory T Cells Which Modulate the Immune Response to Gene Therapy Derived Products in Immunocompetent Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.008  0.429
2005 Biffi A, Capotondo A, Quattrini A, Carro UD, Brambilla R, Fasano S, Marchesini S, Bordignon C, Naldini L. 426. Cure of Established Disease and Direct Evidence of Cellular Cross-Correction in the Nervous System of Metachromatic Leukodystrophy Mice after HSC-Based Gene Therapy Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.428  0.4
2005 Lombardo A, Calos M, Naldini L. 392. Development of a New Lentiviral Vector for Site-Specific Integration into Mammalian Genomes Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.395  0.482
2005 Montini E, Cesana D, Sanvito F, Lucia SS, Fabrizio B, Maurilio P, Doglioni C, Naldini L. 389. Testing the Oncogenic Potential of Retroviral and Lentiviral Vector Integration Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.392  0.473
2005 Arena S, Vigna E, Naldini L, Bardelli A. 373. Use of Lentiviral and Adeno-Associated Vectors for Targeted Gene Inactivation in Human Cancer Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.376  0.415
2005 Copreni E, Castellani S, Palmieri L, Carrabino S, Naldini L, Conese M. 358. Gene Transfer to the Airway Epithelium Mediated by a Third Generation HIV-1 Based Vector: Efficiency and Role of Heparan Sulfate|[ast]| Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.362  0.444
2005 Lombardo A, Beausejour C, Urnov FD, Miller JC, Holmes MC, Gregory PD, Naldini L. 346. Gene Correction of X-Linked SCID Using Engineered Zinc Finger Nucleases and Integration Defective Lentiviral Delivery Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.349  0.482
2005 Dupré L, Scaramuzza S, Marangoni F, Trifari S, Martino S, Tsuchiya S, Thrasher A, Galy A, Naldini L, Aiuti A, Roncarolo MG. 345. Gene Therapy for Wiskott-Aldrich Syndrome Using Lentiviral Vectors: Evidence for Efficacy and Safety after Transduction of Human T Cells and Hematopoietic Stem Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.348  0.441
2005 Mortellaro A, Hernández RJ, Guerrini M, Tabucchi A, Carlucci F, Follenzi A, Naldini L, Bordignon C, Roncarolo MG, Aiuti A. 331. Lentivirus-Mediated Ex Vivo Gene Therapy in ADA-Deficient SCID Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.334  0.471
2005 Marangoni F, Dupré L, Scaramuzza S, Trifari S, Hernández RJ, Thrasher A, Galy A, Aiuti A, Naldini L, Roncarolo MG. 330. Lentiviral Vector Mediated Hematopoietic Stem Cell Gene Therapy Combined with Non-Lethal Conditioning Restores T Cell Function in the Murine Model of Wiskott-Aldrich Syndrome Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.333  0.496
2005 Brown BD, Hauben E, Lombardo A, Sergi LS, Roncarolo MG, Naldini L. 36. Improved Lentiviral Vectors for Systemic Gene Transfer in the Absence of an Immune Response Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.042  0.433
2005 Dupré L, Trifari S, Follenzi A, Marangoni F, Lera TLd, Bernad A, Martino S, Tsuchiya S, Bordignon C, Naldini L, Aiuti A, Roncarolo MG. Erratum to |[ldquo]|Lentiviral Vector-Mediated Gene Transfer in T Cells from Wiskott|[ndash]|Aldrich Syndrome Patients Leads to Functional Correction|[rdquo]| Molecular Therapy. 11: 492. DOI: 10.1016/J.Ymthe.2004.11.013  0.369
2004 Mazzone M, Basilico C, Cavassa S, Pennacchietti S, Risio M, Naldini L, Comoglio PM, Michieli P. An uncleavable form of pro-scatter factor suppresses tumor growth and dissemination in mice Journal of Clinical Investigation. 114: 1418-1432. PMID 15545993 DOI: 10.1172/Jci22235  0.328
2004 Dupré L, Trifari S, Follenzi A, Marangoni F, Lain de Lera T, Bernad A, Martino S, Tsuchiya S, Bordignon C, Naldini L, Aiuti A, Roncarolo MG. Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction Molecular Therapy. 10: 903-915. PMID 15509508 DOI: 10.1016/J.Ymthe.2004.08.008  0.477
2004 Consiglio A, Gritti A, Dolcetta D, Follenzi A, Bordignon C, Gage FH, Vescovi AL, Naldini L. Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors. Proceedings of the National Academy of Sciences of the United States of America. 101: 14835-40. PMID 15466696 DOI: 10.1073/Pnas.0404180101  0.432
2004 Michieli P, Mazzone M, Basilico C, Cavassa S, Sottile A, Naldini L, Comoglio PM. Targeting the tumor and its microenvironment by a dual-function decoy Met receptor Cancer Cell. 6: 61-73. PMID 15261142 DOI: 10.1016/J.Ccr.2004.05.032  0.34
2004 Serafini M, Naldini L, Introna M. Molecular evidence of inefficient transduction of proliferating human B lymphocytes by VSV-pseudotyped HIV-1-derived lentivectors Virology. 325: 413-424. PMID 15246279 DOI: 10.1016/J.Virol.2004.04.038  0.384
2004 Biffi A, De Palma M, Quattrini A, Del Carro U, Amadio S, Visigalli I, Sessa M, Fasano S, Brambilla R, Marchesini S, Bordignon C, Naldini L. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. The Journal of Clinical Investigation. 113: 1118-29. PMID 15085191 DOI: 10.1172/Jci19205  0.48
2004 Follenzi A, Battaglia M, Lombardo A, Annoni A, Roncarolo MG, Naldini L. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice Blood. 103: 3700-3709. PMID 14701690 DOI: 10.1182/Blood-2003-09-3217  0.47
2004 Quattrini A, Biffi A, Amadio S, Bertani I, Dina G, Carro UD, Previtali S, Bordignon C, Naldini L. Correction of metachromatic leukodystrophies (MLD) in the mouse model by transplantation of genetically modified hematopoietic stem cells Journal of the Peripheral Nervous System. 9: 113-114. DOI: 10.1111/J.1085-9489.2004.009209Af.X  0.443
2004 Dupré L, Marangoni F, Follenzi A, Trifari S, Martino S, Tsuchiya S, Bordignon C, Naldini L, Aiuti A, Roncarolo MG. 906. Successful functional correction of T cells from Wiskott-Aldrich syndrome patients with lentiviral vectors Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.812  0.442
2004 Lowenstein PR, Abordo-Adesida E, Barcia C, Follenzi A, Naldini L, Castro MG. 661. Immune System Regulation of Transgene Expression in the Brain 3: Effects of the Immune Response on Transgene Expression from HIV-Derived Lentiviral Vectors Injected into the Rat Striatum Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.607  0.391
2004 Annoni A, Battaglia M, Follenzi A, Lombardo A, Naldini L, Roncarolo M. 631. Modulation of Immune Response to Gene Therapy Derived Products by CD4+ CD25+ T Regulatory Cells Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.561  0.466
2004 Amendola M, Venneri MA, Vigna E, Naldini L. 332. Dual Gene Transfer and Transgenesis by Novel Lentiviral Vectors Carrying Synthetic Bi-Directional Promoters Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.274  0.506
2004 Montini E, Cesana D, Naldini L. 3. Effect of Retroviral and Lentiviral Vector Integrations on Transcription of Flanking Genes Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.084  0.448
2004 Biffi A, Visigalli I, Quattrini A, Bertani I, Palma MD, Carro UD, Brambilla R, Marchesini S, Bordignon C, Naldini L. 41. Ex Vivo Gene Therapy Provides a Unique Therapeutic Benefit and Corrects Metachromatic (MLD) and Globoid (GLD) Leukodystrophy in the Mouse Models Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.064  0.43
2004 Palma MD, Montini E, Sio FRSd, Gentile A, Medico E, Naldini L. 2. Promoter Trapping Reveals Significant Differences in Integration Site Selection Between MLV and HIV Vectors Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.043  0.413
2003 Ruggiero T, Olivero M, Follenzi A, Naldini L, Calogero R, Di Renzo MF. Deletion in a (T)8 microsatellite abrogates expression regulation by 3′-UTR Nucleic Acids Research. 31: 6561-6569. PMID 14602915 DOI: 10.1093/Nar/Gkg858  0.359
2003 De Palma M, Venneri MA, Naldini L. In vivo targeting of tumor endothelial cells by systemic delivery of lentiviral vectors Human Gene Therapy. 14: 1193-1206. PMID 12908970 DOI: 10.1089/104303403322168028  0.451
2003 De Palma M, Venneri MA, Roca C, Naldini L. Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells Nature Medicine. 9: 789-795. PMID 12740570 DOI: 10.1038/Nm871  0.43
2003 Bonci D, Cittadini A, Latronico MVG, Borello U, Aycock JK, Drusco A, Innocenzi A, Follenzi A, Lavitrano M, Monti MG, Ross J, Naldini L, Peschle C, Cossu G, Condorelli G. 'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo Gene Therapy. 10: 630-636. PMID 12692591 DOI: 10.1038/Sj.Gt.3301936  0.309
2003 Cavalieri S, Cazzaniga S, Geuna M, Magnani Z, Bordignon C, Naldini L, Bonini C. Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence Blood. 102: 497-505. PMID 12649146 DOI: 10.1182/Blood-2003-01-0297  0.365
2003 Janssens W, Chuah MKL, Naldini L, Follenzi A, Collen D, Saint-Remy JM, VandenDriessche T. Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent Human Gene Therapy. 14: 263-276. PMID 12639306 DOI: 10.1089/10430340360535814  0.424
2003 Merle U, Encke J, Naldini L, Stremmel W. 1041 Gene therapy of Wilson disease with lentiviral vectors in a rat model Hepatology. 38: 657-657. DOI: 10.1016/S0270-9139(03)81079-0  0.373
2002 Piacibello W, Bruno S, Sanavio F, Droetto S, Gunetti M, Ailles L, De Sio FS, Viale A, Gammaitoni L, Lombardo A, Naldini L, Aglietta M. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice Blood. 100: 4391-4400. PMID 12453876 DOI: 10.1182/Blood.V100.13.4391  0.411
2002 Ailles L, Schmidt M, Santoni De Sio FR, Glimm H, Cavalieri S, Bruno S, Piacibello W, Von Kalle C, Naldini L. Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells Molecular Therapy. 6: 615-626. PMID 12409260 DOI: 10.1006/Mthe.2002.0720  0.493
2002 Manganini M, Rambaldi A, Biondi A, Golay J, Introna M, Serafini M, Bambacioni F, Casati C, Erba E, Follenzi A, Naldini L, Bernasconi S, Gaipa G. A human immunodeficiency virus type 1 pol gene-derived sequence (cPPT/CTS) increases the efficiency of transduction of human nondividing monocytes and T lymphocytes by lentiviral vectors Human Gene Therapy. 13: 1793-1807. PMID 12396613 DOI: 10.1089/104303402760372909  0.413
2002 Horn PA, Morris JC, Bukovsky AA, Andrews RG, Naldini L, Kurre P, Kiem HP. Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons Gene Therapy. 9: 1464-1471. PMID 12378409 DOI: 10.1038/Sj.Gt.3301820  0.398
2002 VandenDriessche T, Thorrez L, Naldini L, Follenzi A, Moons L, Berneman Z, Collen D, Chuah MKL. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo Blood. 100: 813-822. PMID 12130491 DOI: 10.1182/Blood.V100.3.813  0.463
2002 Villani GRD, Follenzi A, Vanacore B, Di Domenico C, Naldini L, Di Natale P. Correction of mucopolysaccharidosis type IIIB fibroblasts by lentiviral vector-mediated gene transfer Biochemical Journal. 364: 747-753. PMID 12049639 DOI: 10.1042/Bj20011872  0.406
2002 Di Natale P, Di Domenico C, Villani GRD, Lombardo A, Follenzi A, Naldini L. In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors European Journal of Biochemistry. 269: 2764-2771. PMID 12047386 DOI: 10.1046/J.1432-1033.2002.02951.X  0.44
2002 Lotti F, Menguzzato E, Rossi C, Naldini L, Ailles L, Mavilio F, Ferrari G. Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement Journal of Virology. 76: 3996-4007. PMID 11907239 DOI: 10.1128/Jvi.76.8.3996-4007.2002  0.523
2002 Ailles LE, Naldini L. HIV-1-derived lentiviral vectors Current Topics in Microbiology and Immunology. 261: 31-52. PMID 11892252 DOI: 10.1007/978-3-642-56114-6_2  0.401
2002 VandenDriessche T, Naldini L, Collen D, Chuah MKL. Oncoretroviral and lentiviral vector-mediated gene therapy Methods in Enzymology. 346: 573-589. PMID 11883092 DOI: 10.1016/S0076-6879(02)46078-8  0.487
2002 De Palma M, Naldini L. Transduction of a gene expression cassette using advanced generation lentiviral vectors Methods in Enzymology. 346: 514-529. PMID 11883088 DOI: 10.1016/S0076-6879(02)46074-0  0.396
2002 Vigna E, Cavalieri S, Ailles L, Geuna M, Loew R, Bujard H, Naldini L. Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors Molecular Therapy. 5: 252-261. PMID 11863414 DOI: 10.1006/Mthe.2002.0542  0.514
2002 Follenzi A, Sabatino G, Lombardo A, Boccaccio C, Naldini L. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors Human Gene Therapy. 13: 243-260. PMID 11812281 DOI: 10.1089/10430340252769770  0.446
2001 Shichinohe T, Bochner BH, Mizutani K, Nishida M, Hegerich-Gilliam S, Naldini L, Kasahara N. Development of lentiviral vectors for antiangiogenic gene delivery Cancer Gene Therapy. 8: 879-889. PMID 11773978 DOI: 10.1038/Sj.Cgt.7700388  0.488
2001 Farson D, Witt R, McGuinness R, Dull T, Kelly M, Song J, Radeke R, Bukovsky A, Consiglio A, Naldini L. A new-generation stable inducible packaging cell line for lentiviral vectors Human Gene Therapy. 12: 981-997. PMID 11387062 DOI: 10.1089/104303401750195935  0.453
2001 Consiglio A, Quattrini A, Martino S, Bensadoun JC, Dolcetta D, Trojani A, Benaglia G, Marchesini S, Cestari V, Oliverio A, Bordignon C, Naldini L. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nature Medicine. 7: 310-6. PMID 11231629 DOI: 10.1038/85454  0.424
2001 Galimi F, Cottone E, Vigna E, Arena N, Boccaccio C, Giordano S, Naldini L, Comoglio PM. Hepatocyte growth factor is a regulator of monocyte-macrophage function Journal of Immunology. 166: 1241-1247. PMID 11145707 DOI: 10.4049/Jimmunol.166.2.1241  0.327
2001 Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nature Medicine. 7: 33-40. PMID 11135613 DOI: 10.1038/83324  0.447
2000 Naldini L, Verma IM. Lentiviral vectors. Advances in Virus Research. 55: 599-609. PMID 11050959  0.443
2000 Vigna E, Naldini L. Lentiviral vectors: Excellent tools for experimental gene transfer and promising candidates for gene therapy Journal of Gene Medicine. 2: 308-316. PMID 11045424 DOI: 10.1002/1521-2254(200009/10)2:5<308::Aid-Jgm131>3.0.Co;2-3  0.461
2000 Guenechea G, Gan OI, Inamitsu T, Dorrell C, Pereira DS, Kelly M, Naldini L, Dick JE. Transduction of human CD34+CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors Molecular Therapy. 1: 566-573. PMID 10933981 DOI: 10.1006/Mthe.2000.0077  0.443
2000 Follenzi A, Ailles LE, Bakovic S, Geuna M, Naldini L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences Nature Genetics. 25: 217-222. PMID 10835641 DOI: 10.1038/76095  0.449
2000 Johnson LG, Olsen JC, Naldini L, Boucher RC. Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo. Gene Therapy. 7: 568-74. PMID 10819571 DOI: 10.1038/Sj.Gt.3301138  0.323
2000 Park F, Ohashi K, Chiu W, Naldini L, Mark A K. Efficient lentiviral transduction of liver requires cell cycling in vivo Nature Genetics. 24: 49-52. PMID 10615126 DOI: 10.1038/71673  0.425
1999 Naldini L. In vivo gene delivery by lentiviral vectors Thrombosis and Haemostasis. 82: 552-554. PMID 10605750 DOI: 10.1055/S-0037-1615879  0.468
1999 Bukovsky AA, Song JP, Naldini L. Interaction of human immunodeficiency virus-derived vectors with wild- type virus in transduced cells Journal of Virology. 73: 7087-7092. PMID 10400815 DOI: 10.1128/Jvi.73.8.7087-7092.1999  0.319
1999 Case SS, Price MA, Jordan CT, Yu XJ, Wang L, Bauer G, Haas DL, Xu D, Stripecke R, Naldini L, Kohn DB, Crooks GM. Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proceedings of the National Academy of Sciences of the United States of America. 96: 2988-93. PMID 10077624 DOI: 10.1073/Pnas.96.6.2988  0.395
1999 Naldini L, Verma IM. 3 Lentiviral Vectors Cold Spring Harbor Monograph Archive. 36: 47-60. DOI: 10.1101/087969528.36.47  0.623
1998 Zufferey R, Dull T, Mandel RJ, Bukovsky A, Quiroz D, Naldini L, Trono D. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. Journal of Virology. 72: 9873-80. PMID 9811723 DOI: 10.1128/Jvi.72.12.9873-9880.1998  0.39
1998 Dull T, Zufferey R, Kelly M, Mandel RJ, Nguyen M, Trono D, Naldini L. A third-generation lentivirus vector with a conditional packaging system. Journal of Virology. 72: 8463-71. PMID 9765382 DOI: 10.1128/Jvi.72.11.8463-8471.1998  0.41
1998 Kelly M, Naldini L. Lentiviral vectors for gene therapy Biogenic Amines. 14: 425-432.  0.307
1997 Zufferey R, Nagy D, Mandel RJ, Naldini L, Trono D. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nature Biotechnology. 15: 871-5. PMID 9306402 DOI: 10.1038/Nbt0997-871  0.469
1997 Blömer U, Naldini L, Kafri T, Trono D, Verma IM, Gage FH. Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. Journal of Virology. 71: 6641-9. PMID 9261386 DOI: 10.1128/Jvi.71.9.6641-6649.1997  0.586
1997 Blömer U, Kafri T, Naldini L, Verma I, Gage F. Efficient and sustained gene transfer in terminally differentiated cells with a lentiviral vector European Journal of Cancer. 33: S51. DOI: 10.1016/S0959-8049(97)89458-0  0.594
1996 Naldini L, Blömer U, Gage FH, Trono D, Verma IM. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proceedings of the National Academy of Sciences of the United States of America. 93: 11382-8. PMID 8876144 DOI: 10.1073/Pnas.93.21.11382  0.571
1996 Blömer U, Naldini L, Verma IM, Trono D, Gage FH. Applications of gene therapy to the CNS. Human Molecular Genetics. 5: 1397-404. PMID 8875243 DOI: 10.1093/Hmg/5.Supplement_1.1397  0.591
1996 Naldini L, Blömer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science (New York, N.Y.). 272: 263-7. PMID 8602510 DOI: 10.1126/Science.272.5259.263  0.596
1995 Naldini L, Vigna E, Bardelli A, Follenzi A, Galimi F, Comoglio PM. Biological activation of pro-HGF (hepatocyte growth factor) by urokinase is controlled by a stoichiometric reaction Journal of Biological Chemistry. 270: 603-611. PMID 7822285 DOI: 10.1074/Jbc.270.2.603  0.318
1994 Gaudino G, Follenzi A, Naldini L, Collesi C, Santoro M, Gallo KA, Godowski PJ, Comoglio PM. Ron is a heterodimeric tyrosine kinase receptor activated by the HGF homologue MSP Embo Journal. 13: 3524-3532. PMID 8062829 DOI: 10.1002/J.1460-2075.1994.Tb06659.X  0.315
1992 Bussolino F, Renzo MFD, Ziche M, Bocchietto E, Olivero M, Naldini L, Gaudino G, Tamagnone L, Coffer A, Comoglio PM. Hepatocyte growth factor is a potent angiogenic factor which stimulates endothelial cell motility and growth Journal of Cell Biology. 119: 629-641. PMID 1383237 DOI: 10.1083/Jcb.119.3.629  0.342
1991 Naldini L, Weidner KM, Vigna E, Gaudino G, Bardelli A, Ponzetto C, Narsimhan RP, Hartmann G, Zarnegar R, Michalopoulos GK. Scatter factor and hepatocyte growth factor are indistinguishable ligands for the MET receptor. The Embo Journal. 10: 2867-2878. DOI: 10.1002/J.1460-2075.1991.Tb07836.X  0.314
1988 Gaudino G, Cirillo D, Naldini L, Rossino P, Comoglio PM. Activation of the protein-tyrosine kinase associated with the bombesin receptor complex in small cell lung carcinomas Proceedings of the National Academy of Sciences of the United States of America. 85: 2166-2170. PMID 2451242 DOI: 10.1073/Pnas.85.7.2166  0.301
1986 Naldini L, Stacchini A, Cirillo DM, Aglietta M, Gavosto F, Comoglio PM. Phosphotyrosine antibodies identify the p210c-abl tyrosine kinase and proteins phosphorylated on tyrosine in human chronic myelogenous leukemia cells. Molecular and Cellular Biology. 6: 1803-1811. PMID 2431286 DOI: 10.1128/Mcb.6.5.1803  0.33
1984 Cirillo D, Comoglio PM, Naldini L, Marchisio PC, Ronchetti IP. Visualization of phosphotyrosine containing molecules within the detergent insoluble cell matrix of v-src transformed fibroblasts Cell Biology International Reports. 8: 959-969. PMID 6210153 DOI: 10.1016/0309-1651(84)90194-2  0.308
Show low-probability matches.