| Year |
Citation |
Score |
| 2023 |
Kumar P, Wang M, Kumru OS, Hickey JM, Sanmiguel J, Zabaleta N, Vandenberghe LH, Joshi SB, Volkin DB. Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate. Molecular Therapy. Methods & Clinical Development. 30: 103-121. PMID 37746246 DOI: 10.1016/j.omtm.2023.06.004 |
0.743 |
|
| 2023 |
Reichart D, Newby GA, Wakimoto H, Lun M, Gorham JM, Curran JJ, Raguram A, DeLaughter DM, Conner DA, Marsiglia JDC, Kohli S, Chmatal L, Page DC, Zabaleta N, Vandenberghe L, et al. Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. Nature Medicine. PMID 36797483 DOI: 10.1038/s41591-022-02190-7 |
0.732 |
|
| 2022 |
Hsu Y, Bhattarai S, Thompson JM, Mahoney A, Thomas J, Mayer SK, Datta P, Garrison J, Searby CC, Vandenberghe LH, Seo S, Sheffield VC, Drack AV. Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model. Molecular Therapy. Nucleic Acids. 31: 164-181. PMID 36700052 DOI: 10.1016/j.omtn.2022.12.007 |
0.315 |
|
| 2022 |
Florea M, Nicolaou F, Pacouret S, Zinn EM, Sanmiguel J, Andres-Mateos E, Unzu C, Wagers AJ, Vandenberghe LH. High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography. Molecular Therapy. Methods & Clinical Development. 28: 146-159. PMID 36654797 DOI: 10.1016/j.omtm.2022.12.009 |
0.734 |
|
| 2022 |
Zinn E, Unzu C, Schmit PF, Turunen HT, Zabaleta N, Sanmiguel J, Fieldsend A, Bhatt U, Diop C, Merkel E, Gurrala R, Peacker B, Rios C, Messemer K, Santos J, ... ... Vandenberghe LH, et al. Ancestral library identifies conserved reprogrammable liver motif on AAV capsid. Cell Reports. Medicine. 100803. PMID 36327973 DOI: 10.1016/j.xcrm.2022.100803 |
0.801 |
|
| 2022 |
Lu YC, Tsai YH, Chan YH, Hu CJ, Huang CY, Xiao R, Hsu CJ, Vandenberghe LH, Wu CC, Cheng YF. Gene therapy with a synthetic adeno-associated viral vector improves audiovestibular phenotypes in Pjvk-mutant mice. Jci Insight. 7. PMID 36278489 DOI: 10.1172/jci.insight.152941 |
0.321 |
|
| 2022 |
Zabaleta N, Bhatt U, Hérate C, Maisonnasse P, Sanmiguel J, Diop C, Castore S, Estelien R, Li D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Pascal Q, Naninck T, Kahlaoui N, ... ... Vandenberghe LH, et al. Durable immunogenicity, adaptation to emerging variants and low dose efficacy of AAV-based COVID19 platform in macaques. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 35546782 DOI: 10.1016/j.ymthe.2022.05.007 |
0.754 |
|
| 2022 |
Maciorowski D, Diop C, Bhatt U, Estelien R, Li D, Chauhan R, Vandenberghe LH, Zabaleta N. Immunogenicity of an AAV-Based COVID-19 Vaccine in Murine Models of Obesity and Aging. Viruses. 14. PMID 35458550 DOI: 10.3390/v14040820 |
0.706 |
|
| 2022 |
Naninck T, Kahlaoui N, Lemaitre J, Maisonnasse P, De Mori A, Pascal Q, Contreras V, Marlin R, Relouzat F, Delache B, Hérate C, Aldon Y, van Gils M, Zabaleta N, Ho Tsong Fang R, ... ... Vandenberghe LH, et al. Computed tomography and [F]-FDG PET imaging provide additional readouts for COVID-19 pathogenesis and therapies evaluation in non-human primates. Iscience. 25: 104101. PMID 35313622 DOI: 10.1016/j.isci.2022.104101 |
0.694 |
|
| 2022 |
Andres-Mateos E, Landegger LD, Unzu C, Phillips J, Lin BM, Dewyer NA, Sanmiguel J, Nicolaou F, Valero MD, Bourdeu KI, Sewell WF, Beiler RJ, McKenna MJ, Stankovic KM, Vandenberghe LH. Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate. Nature Communications. 13: 1359. PMID 35292639 DOI: 10.1038/s41467-022-28969-3 |
0.39 |
|
| 2022 |
Grotz S, Schäfer J, Wunderlich KA, Ellederova Z, Auch H, Bähr A, Runa-Vochozkova P, Fadl J, Arnold V, Ardan T, Veith M, Santamaria G, Dhom G, Hitzl W, Kessler B, ... ... Vandenberghe LH, et al. Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes. Embo Molecular Medicine. e14817. PMID 35254721 DOI: 10.15252/emmm.202114817 |
0.319 |
|
| 2021 |
Katz MG, Hadas Y, Bailey RA, Fazal S, Vincek A, Madjarova SJ, Shtraizent N, Vandenberghe LH, Eliyahu E. Efficient cardiac gene transfer and early-onset expression of a synthetic adeno-associated viral vector, Anc80L65, after intramyocardial administration. The Journal of Thoracic and Cardiovascular Surgery. PMID 34985414 DOI: 10.1016/j.jtcvs.2021.05.050 |
0.377 |
|
| 2021 |
Ilyinskii PO, Michaud AM, Rizzo GL, Roy CJ, Leung SS, Elkins SL, Capela T, Chowdhury A, Li L, Chandler RJ, Manoli I, Andres-Mateos E, Johnston LPM, Vandenberghe LH, Venditti CP, et al. ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia. Molecular Therapy. Methods & Clinical Development. 22: 279-292. PMID 34485611 DOI: 10.1016/j.omtm.2021.06.015 |
0.315 |
|
| 2021 |
Zabaleta N, Dai W, Bhatt U, Hérate C, Maisonnasse P, Chichester JA, Sanmiguel J, Estelien R, Michalson KT, Diop C, Maciorowski D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Naninck T, ... ... Vandenberghe LH, et al. An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates. Cell Host & Microbe. PMID 34428428 DOI: 10.1016/j.chom.2021.08.002 |
0.796 |
|
| 2021 |
De Rosa S, Salani M, Smith S, Sangster M, Miller-Browne V, Wassmer S, Xiao R, Vandenberghe L, Slaugenhaupt S, Misko A, Grishchuk Y. MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV. Human Molecular Genetics. PMID 33822942 DOI: 10.1093/hmg/ddab093 |
0.338 |
|
| 2021 |
Zabaleta N, Dai W, Bhatt U, Chichester JA, Estelien R, Sanmiguel J, Michalson KT, Diop C, Maciorowski D, Qi W, Hudspeth E, Cucalon A, Dyer CD, Pampena MB, Knox JJ, ... ... Vandenberghe LH, et al. Immunogenicity of an AAV-based, room-temperature stable, single dose COVID-19 vaccine in mice and non-human primates. Biorxiv : the Preprint Server For Biology. PMID 33442684 DOI: 10.1101/2021.01.05.422952 |
0.757 |
|
| 2020 |
Jacob A, Brun L, Gil PJ, Ménard L, Bouzelha M, Broucque F, Roblin A, Vandenberghe LH, Adjali O, Robin C, François A, Blouin V, Penaud-Budloo M, Ayuso E. Homologous Recombination Offers Advantages Over Transposition-Based Systems to Generate Recombinant Baculovirus for Adeno-Associated Viral Vector Production. Biotechnology Journal. e2000014. PMID 33067902 DOI: 10.1002/biot.202000014 |
0.349 |
|
| 2020 |
Greenwald SH, Brown EE, Scandura MJ, Hennessey E, Farmer R, Pawlyk BS, Xiao R, Vandenberghe LH, Pierce EA. Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of -Associated Retinal Degeneration. Molecular Therapy. Methods & Clinical Development. 18: 582-594. PMID 32775493 DOI: 10.1016/J.Omtm.2020.07.003 |
0.454 |
|
| 2020 |
Hu CJ, Lu YC, Tsai YH, Cheng HY, Takeda H, Huang CY, Xiao R, Hsu CJ, Tsai JW, Vandenberghe LH, Wu CC, Cheng YF. Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65. Molecular Therapy. Methods & Clinical Development. 18: 493-500. PMID 32775487 DOI: 10.1016/J.Omtm.2020.06.019 |
0.482 |
|
| 2020 |
Omichi R, Yoshimura H, Shibata SB, Vandenberghe LH, Smith RJH. Hair Cell Transduction Efficiency of Single- and Dual-AAV Serotypes in Adult Murine Cochleae. Molecular Therapy. Methods & Clinical Development. 17: 1167-1177. PMID 32518805 DOI: 10.1016/J.Omtm.2020.05.007 |
0.409 |
|
| 2020 |
Vandenberghe LH. COVID-19: Gene Transfer to the Rescue? Human Gene Therapy. PMID 32469280 DOI: 10.1089/Hum.2020.29125.Lhv |
0.364 |
|
| 2020 |
Schmit PF, Pacouret S, Zinn E, Telford E, Nicolaou F, Broucque F, Andres-Mateos E, Xiao R, Penaud-Budloo M, Bouzelha M, Jaulin N, Adjali O, Ayuso E, Vandenberghe LH. Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries. Molecular Therapy. Methods & Clinical Development. 17: 107-121. PMID 31909084 DOI: 10.1016/J.Omtm.2019.11.014 |
0.732 |
|
| 2019 |
Dudek AM, Zabaleta N, Zinn E, Pillay S, Zengel J, Porter C, Franceschini JS, Estelien R, Carette JE, Zhou GL, Vandenberghe LH. GPR108 Is a Highly Conserved AAV Entry Factor. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 31784416 DOI: 10.1016/J.Ymthe.2019.11.005 |
0.812 |
|
| 2019 |
Subramanian S, Maurer A, Bator C, Makhov A, Conway J, Turner KB, Marden J, Vandenberghe LH, Hafenstein S. Filling AAV capsids: estimating success by cryo-EM. Human Gene Therapy. PMID 31530236 DOI: 10.1089/Hum.2019.041 |
0.354 |
|
| 2019 |
Goldstein JM, Tabebordbar M, Zhu K, Wang LD, Messemer KA, Peacker B, Ashrafi Kakhki S, Gonzalez-Celeiro M, Shwartz Y, Cheng JKW, Xiao R, Barungi T, Albright C, Hsu YC, Vandenberghe LH, et al. In Situ Modification of Tissue Stem and Progenitor Cell Genomes. Cell Reports. 27: 1254-1264.e7. PMID 31018138 DOI: 10.1016/J.Celrep.2019.03.105 |
0.326 |
|
| 2019 |
Hudry E, Vandenberghe LH. Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality. Neuron. 101: 839-862. PMID 30844402 DOI: 10.1016/J.Neuron.2019.02.017 |
0.429 |
|
| 2019 |
Sanmiguel J, Gao G, Vandenberghe LH. Quantitative and Digital Droplet-Based AAV Genome Titration. Methods in Molecular Biology (Clifton, N.J.). 1950: 51-83. PMID 30783968 DOI: 10.1007/978-1-4939-9139-6_4 |
0.428 |
|
| 2019 |
Maurer AC, Cepeda Diaz AK, Vandenberghe LH. Residues on AAV Capsid Lumen Dictate Interactions and Compatibility With the Assembly-activating Protein. Journal of Virology. PMID 30651367 DOI: 10.1128/Jvi.02013-18 |
0.345 |
|
| 2019 |
Vandenberghe LH. Addgene: Molecular Therapy Interview with Melina Fan and Karen Guerin Molecular Therapy. 27: 6-9. DOI: 10.1016/J.Ymthe.2018.12.001 |
0.392 |
|
| 2018 |
Shen J, Xiao R, Bair J, Wang F, Vandenberghe LH, Dartt D, Baranov P, Ng YSE. Novel engineered, membrane-localized variants of vascular endothelial growth factor (VEGF) protect retinal ganglion cells: a proof-of-concept study. Cell Death & Disease. 9: 1018. PMID 30282966 DOI: 10.1038/S41419-018-1049-0 |
0.339 |
|
| 2018 |
Hudry E, Andres-Mateos E, Lerner EP, Volak A, Cohen O, Hyman BT, Maguire CA, Vandenberghe LH. Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65. Molecular Therapy. Methods & Clinical Development. 10: 197-209. PMID 30109242 DOI: 10.1016/J.Omtm.2018.07.006 |
0.687 |
|
| 2018 |
Miller JW, Vandenberghe LH. Breaking and Sealing Barriers in Retinal Gene Therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 30107998 DOI: 10.1016/J.Ymthe.2018.08.003 |
0.415 |
|
| 2018 |
Ikeda Y, Sun Z, Ru X, Vandenberghe LH, Humphreys BD. Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector. Journal of the American Society of Nephrology : Jasn. PMID 29976586 DOI: 10.1681/Asn.2018040426 |
0.38 |
|
| 2018 |
Maurer AC, Pacouret S, Cepeda Diaz AK, Blake J, Andres-Mateos E, Vandenberghe LH. The Assembly-Activating Protein Promotes Stability and Interactions between AAV's Viral Proteins to Nucleate Capsid Assembly. Cell Reports. 23: 1817-1830. PMID 29742436 DOI: 10.1016/J.Celrep.2018.04.026 |
0.405 |
|
| 2018 |
Dudek AM, Pillay S, Puschnik AS, Nagamine CM, Cheng F, Qiu J, Carette JE, Vandenberghe LH. An alternate route for adeno-associated virus entry independent of AAVR. Journal of Virology. PMID 29343568 DOI: 10.1128/Jvi.02213-17 |
0.517 |
|
| 2018 |
Carvalho LS, Xiao R, Wassmer S, Langsdorf A, Zinn E, Pacouret S, Shah S, Comander JI, Kim L, Lim L, Vandenberghe LH. Synthetic adeno-associated viral vector efficiently targets mouse and non-human primate retina in vivo. Human Gene Therapy. PMID 29325457 DOI: 10.1089/Hum.2017.154 |
0.812 |
|
| 2017 |
Tao Y, Huang M, Shu Y, Ruprecht A, Wang H, Tang Y, Vandenberghe LH, Wang Q, Gao G, Kong WJ, Chen ZY. Delivery of Adeno-Associated Viral Vectors in Adult Mammalian Inner Ear Cell Subtypes without Auditory Dysfunction. Human Gene Therapy. PMID 29130354 DOI: 10.1089/Hum.2017.120 |
0.426 |
|
| 2017 |
Carvalho LS, Turunen HT, Wassmer SJ, Luna-Velez MV, Xiao R, Bennett J, Vandenberghe LH. Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting. Frontiers in Neuroscience. 11: 503. PMID 28943836 DOI: 10.3389/Fnins.2017.00503 |
0.728 |
|
| 2017 |
Wang L, Xiao R, Andres-Mateos E, Vandenberghe LH. Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye. Plos One. 12: e0182473. PMID 28763501 DOI: 10.1371/Journal.Pone.0182473 |
0.491 |
|
| 2017 |
Huang X, Zhou G, Wu W, Duan Y, Ma G, Song J, Xiao R, Vandenberghe L, Zhang F, D'Amore PA, Lei H. Genome editing abrogates angiogenesis in vivo. Nature Communications. 8: 112. PMID 28740073 DOI: 10.1038/S41467-017-00140-3 |
0.329 |
|
| 2017 |
Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC. Corrigendum: Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Scientific Reports. 7: 46827. PMID 28530226 DOI: 10.1038/Srep46827 |
0.38 |
|
| 2017 |
Pacouret S, Bouzelha M, Shelke R, Andres-Mateos E, Xiao R, Maurer A, Mevel M, Turunen H, Barungi T, Penaud-Budloo M, Broucque F, Blouin V, Moullier P, Ayuso E, Vandenberghe LH. AAV-ID: a Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28427840 DOI: 10.1016/J.Ymthe.2017.04.001 |
0.393 |
|
| 2017 |
Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC. Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Scientific Reports. 7: 45524. PMID 28367981 DOI: 10.1038/Srep45524 |
0.437 |
|
| 2017 |
Wassmer SJ, Carvalho LS, György B, Vandenberghe LH, Maguire CA. Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection. Scientific Reports. 7: 45329. PMID 28361998 DOI: 10.1038/Srep45329 |
0.771 |
|
| 2017 |
Pan B, Askew C, Galvin A, Heman-Ackah S, Asai Y, Indzhykulian AA, Jodelka FM, Hastings ML, Lentz JJ, Vandenberghe LH, Holt JR, Géléoc GS. Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c. Nature Biotechnology. PMID 28165476 DOI: 10.1038/Nbt.3801 |
0.386 |
|
| 2017 |
Landegger LD, Pan B, Askew C, Wassmer SJ, Gluck SD, Galvin A, Taylor R, Forge A, Stankovic KM, Holt JR, Vandenberghe LH. A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear. Nature Biotechnology. PMID 28165475 DOI: 10.1038/Nbt.3781 |
0.493 |
|
| 2016 |
Garanto A, Chung DC, Duijkers L, Corral-Serrano JC, Messchaert M, Xiao R, Bennett J, Vandenberghe LH, Collin RW. In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery. Human Molecular Genetics. PMID 27106101 DOI: 10.1093/Hmg/Ddw118 |
0.538 |
|
| 2016 |
Tabebordbar M, Zhu K, Cheng JK, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science (New York, N.Y.). 351: 407-11. PMID 26721686 DOI: 10.1126/Science.Aad5177 |
0.353 |
|
| 2016 |
Castle MJ, Turunen HT, Vandenberghe LH, Wolfe JH. Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids. Methods in Molecular Biology (Clifton, N.J.). 1382: 133-49. PMID 26611584 DOI: 10.1007/978-1-4939-3271-9_10 |
0.42 |
|
| 2016 |
Carvalho LS, Vandenberghe LH. Understanding Cone Photoreceptor Cell Death in Achromatopsia. Advances in Experimental Medicine and Biology. 854: 231-6. PMID 26427416 DOI: 10.1007/978-3-319-17121-0_31 |
0.535 |
|
| 2016 |
Xiao R, Wang L, Carvalho L, Wassmer S, Vandenberghe LH. 608. In Silico Reconstructed Ancestral Adeno-Associated Viruses Transduce Mouse Anterior Segment Molecular Therapy. 24: S241. DOI: 10.1016/S1525-0016(16)33416-5 |
0.475 |
|
| 2016 |
György B, Wassmer S, Carvalho L, Maguire C, Vandenberghe LH. 594. Exosome-Associated AAV Enhances Retinal Transduction Following Intravitreal Injection Molecular Therapy. 24: S235. DOI: 10.1016/S1525-0016(16)33402-5 |
0.745 |
|
| 2016 |
Tabebordbar M, Zhu K, Cheng J, Widrick J, Yan W, Xiao R, Vandenberghe L, Zhang F, Wagers A. 483. In Vivo DMD Gene Editing in Muscles and Muscle Stem Cells of Dystrophic Mice Molecular Therapy. 24: S191-S192. DOI: 10.1016/S1525-0016(16)33292-0 |
0.359 |
|
| 2016 |
Turunen HT, Zinn E, Vandenberghe LH. 467. Assay Conditions Significantly Alter AAV Neutralizing Antibody Determination Molecular Therapy. 24: S184-S185. DOI: 10.1016/S1525-0016(16)33276-2 |
0.737 |
|
| 2016 |
Landegger L, Pang B, Wassmer S, Gluck S, Galvin A, Stankovic KM, Holt JR, Vandenberghe LH. 269. Novel Synthetic AAV Efficiently Transduces Neurosensory Hair Cells in the Cochlea Molecular Therapy. 24: S107. DOI: 10.1016/S1525-0016(16)33078-7 |
0.401 |
|
| 2016 |
Wassmer S, Comander J, Carvalho L, Xiao R, Plovie E, Langsdorf A, Lim L, Hafler B, Wu DM, Eliott D, Kim LA, Vandenberghe LH. 80. Delayed Inflammatory Response to Intravitreal AAV Gene Transfer in Non-Human Primates Molecular Therapy. 24: S35. DOI: 10.1016/S1525-0016(16)32889-1 |
0.574 |
|
| 2016 |
Pacouret S, Mevel M, Bouzelha M, Maurer A, Shelke R, Xiao R, Penaud-Budloo M, Turunen H, Broucque F, Blouin V, Moullier P, Ayuso E, Vandenberghe LH. 34. Development and Validation of Identity and Homogeneity Assays for AAV Preparations Molecular Therapy. 24: S15. DOI: 10.1016/S1525-0016(16)32843-X |
0.413 |
|
| 2016 |
Comander J, Carvalho L, Wassmer S, Xiao R, Plovie E, Langsdorf A, Lim L, Hafler B, Wu DM, Eliott D, Kim LA, Vandenberghe LH. 29. Novel Surgical Method for Intravitreal AAV Administration Overcomes Transduction Barriers in Non-Human Primates Molecular Therapy. 24: S13-S14. DOI: 10.1016/S1525-0016(16)32838-6 |
0.62 |
|
| 2015 |
Zinn E, Pacouret S, Khaychuk V, Turunen HT, Carvalho LS, Andres-Mateos E, Shah S, Shelke R, Maurer AC, Plovie E, Xiao R, Vandenberghe LH. In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector. Cell Reports. 12: 1056-68. PMID 26235624 DOI: 10.1016/J.Celrep.2015.07.019 |
0.814 |
|
| 2015 |
Vandenberghe LH. What Is Next for Retinal Gene Therapy? Cold Spring Harbor Perspectives in Medicine. PMID 25877395 DOI: 10.1101/Cshperspect.A017442 |
0.44 |
|
| 2015 |
Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, ... ... Vandenberghe LH, et al. Perspectives on best practices for gene therapy programs. Human Gene Therapy. 26: 127-33. PMID 25654329 DOI: 10.1089/Hum.2014.147 |
0.506 |
|
| 2015 |
Carvalho LS, Vandenberghe LH. Promising and delivering gene therapies for vision loss. Vision Research. 111: 124-33. PMID 25094052 DOI: 10.1016/J.Visres.2014.07.013 |
0.595 |
|
| 2015 |
Carvalho LS, Xiao R, Comander JI, Kim LA, Lim L, Khaychuck V, Plovie E, Zinn E, Vandenberghe LH. 383. Ancestral AAV Demonstrates Unique Transduction Properties in Mouse and Primate Molecular Therapy. 23: S152-S153. DOI: 10.1016/S1525-0016(16)33992-2 |
0.804 |
|
| 2015 |
Carvalho LS, György B, Mu D, Shah S, Vandenberghe LH, Maguire CA. 152. Retinal Tropism of Exosome-Associated AAV Vector Via Intravitreal Delivery Molecular Therapy. 23: S61. DOI: 10.1016/S1525-0016(16)33757-1 |
0.725 |
|
| 2015 |
Zinn EM, Khaychuk V, Turunen H, Vandenberghe LH. 89. In Silico Reconstruction of AAV Evolutionary Intermediates To Identify Genetic Determinants of Capsid Function Molecular Therapy. 23: S38. DOI: 10.1016/S1525-0016(16)33694-2 |
0.776 |
|
| 2015 |
Pacouret S, Turunen H, Plovie-Buys E, Xiao R, Shelke R, Lin Y, Zinn E, Vandenberghe LH. 88. Structural Studies Into the Stability of Novel In Silico Reconstructed Ancestral AAV Vectors Molecular Therapy. 23: S38. DOI: 10.1016/S1525-0016(16)33693-0 |
0.77 |
|
| 2014 |
Zinn E, Vandenberghe LH. Adeno-associated virus: fit to serve. Current Opinion in Virology. 8: 90-7. PMID 25128609 DOI: 10.1016/J.Coviro.2014.07.008 |
0.767 |
|
| 2014 |
Cronin T, Vandenberghe LH, Hantz P, Juttner J, Reimann A, Kacsó AE, Huckfeldt RM, Busskamp V, Kohler H, Lagali PS, Roska B, Bennett J. Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter. Embo Molecular Medicine. 6: 1175-90. PMID 25092770 DOI: 10.15252/Emmm.201404077 |
0.801 |
|
| 2014 |
Bell P, Vandenberghe LH, Wilson JM. Formation of newly synthesized adeno-associated virus capsids in the cell nucleus. Human Gene Therapy Methods. 25: 179-80. PMID 24933465 DOI: 10.1089/hgtb.2014.054 |
0.372 |
|
| 2014 |
O'Reilly M, Federoff HJ, Fong Y, Kohn DB, Patterson AP, Ahmed N, Asokan A, Boye SE, Crystal RG, De Oliveira S, Gargiulo L, Harper SQ, Ikeda Y, Jambou R, Montgomery M, ... ... Vandenberghe LH, et al. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Human Gene Therapy. 25: 488-97. PMID 24773122 DOI: 10.1089/Hum.2014.045 |
0.375 |
|
| 2014 |
Mikals K, Nam HJ, Van Vliet K, Vandenberghe LH, Mays LE, McKenna R, Wilson JM, Agbandje-McKenna M. The structure of AAVrh32.33, a novel gene delivery vector. Journal of Structural Biology. 186: 308-17. PMID 24704217 DOI: 10.1016/J.Jsb.2014.03.020 |
0.758 |
|
| 2013 |
Lagor WR, Johnston JC, Lock M, Vandenberghe LH, Rader DJ. Adeno-associated viruses as liver-directed gene delivery vehicles: focus on lipoprotein metabolism. Methods in Molecular Biology (Clifton, N.J.). 1027: 273-307. PMID 23912992 DOI: 10.1007/978-1-60327-369-5_13 |
0.457 |
|
| 2013 |
Simonato M, Bennett J, Boulis NM, Castro MG, Fink DJ, Goins WF, Gray SJ, Lowenstein PR, Vandenberghe LH, Wilson TJ, Wolfe JH, Glorioso JC. Progress in gene therapy for neurological disorders. Nature Reviews. Neurology. 9: 277-91. PMID 23609618 DOI: 10.1038/Nrneurol.2013.56 |
0.55 |
|
| 2013 |
Cepko CL, Vandenberghe LH. Retinal gene therapy coming of age. Human Gene Therapy. 24: 242-4. PMID 23458444 DOI: 10.1089/Hum.2013.050 |
0.356 |
|
| 2013 |
Vandenberghe LH, Bell P, Maguire AM, Xiao R, Hopkins TB, Grant R, Bennett J, Wilson JM. AAV9 targets cone photoreceptors in the nonhuman primate retina. Plos One. 8: e53463. PMID 23382846 DOI: 10.1371/Journal.Pone.0053463 |
0.653 |
|
| 2013 |
Vandenberghe LH, Bell P, Maguire AM, Xiao R, Hopkins TB, Grant R, Bennett J, Wilson JM. Correction: AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina Plos One. 8. DOI: 10.1371/Annotation/64B90996-4634-4C63-B737-634397B0B445 |
0.556 |
|
| 2013 |
Simonato M, Bennett J, Boulis NM, Castro MG, Fink D, Goins WF, Gray SJ, Lowenstein PR, Vandenberghe L, Wilson TJ, Wolfe JH, Glorioso JC. Correction: Progress in gene therapy for neurological disorders Nature Reviews Neurology. 9: 298-298. DOI: 10.1038/Nrneurol.2013.103 |
0.466 |
|
| 2012 |
Holt JR, Vandenberghe LH. Gene therapy for deaf mice goes viral. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1836-7. PMID 23023054 DOI: 10.1038/Mt.2012.196 |
0.431 |
|
| 2012 |
Mason JB, Vandenberghe LH, Xiao R, Wilson JM, Richardson DW. Influence of serotype, cell type, tissue composition, and time after inoculation on gene expression in recombinant adeno-associated viral vector-transduced equine joint tissues. American Journal of Veterinary Research. 73: 1178-85. PMID 22849678 DOI: 10.2460/Ajvr.73.8.1178 |
0.498 |
|
| 2012 |
Vandenberghe LH, Auricchio A. Novel adeno-associated viral vectors for retinal gene therapy. Gene Therapy. 19: 162-8. PMID 21993172 DOI: 10.1038/Gt.2011.151 |
0.67 |
|
| 2012 |
Gurda B, Bell P, Zhu Y, Wang P, O'Donnell P, Sanmiguel J, Vandenberghe L, Haskins M, Wilson J. Liver-Directed Gene Therapy for Mucopolysaccharidosis Type I (MPS I) Molecular Genetics and Metabolism. 105: S32. DOI: 10.1016/J.Ymgme.2011.11.067 |
0.475 |
|
| 2011 |
Aleman TS, Cideciyan AV, Aguirre GK, Huang WC, Mullins CL, Roman AJ, Sumaroka A, Olivares MB, Tsai FF, Schwartz SB, Vandenberghe LH, Limberis MP, Stone EM, Bell P, Wilson JM, et al. Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. Investigative Ophthalmology & Visual Science. 52: 6898-910. PMID 21757580 DOI: 10.1167/Iovs.11-7701 |
0.471 |
|
| 2011 |
Vandenberghe LH, Bell P, Maguire AM, Cearley CN, Xiao R, Calcedo R, Wang L, Castle MJ, Maguire AC, Grant R, Wolfe JH, Wilson JM, Bennett J. Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Science Translational Medicine. 3: 88ra54. PMID 21697530 DOI: 10.1126/Scitranslmed.3002103 |
0.696 |
|
| 2011 |
Kassim SH, Vandenberghe LH, Hovhannisyan R, Wilson JM, Rader DJ. Identification and functional characterization in vivo of a novel splice variant of LDLR in rhesus macaques. Physiological Genomics. 43: 911-6. PMID 21628398 DOI: 10.1152/Physiolgenomics.00006.2011 |
0.777 |
|
| 2011 |
Bell CL, Vandenberghe LH, Bell P, Limberis MP, Gao GP, Van Vliet K, Agbandje-McKenna M, Wilson JM. The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. The Journal of Clinical Investigation. 121: 2427-35. PMID 21576824 DOI: 10.1172/Jci57367 |
0.721 |
|
| 2011 |
Van der Perren A, Toelen J, Carlon M, Van den Haute C, Coun F, Heeman B, Reumers V, Vandenberghe LH, Wilson JM, Debyser Z, Baekelandt V. Efficient and stable transduction of dopaminergic neurons in rat substantia nigra by rAAV 2/1, 2/2, 2/5, 2/6.2, 2/7, 2/8 and 2/9. Gene Therapy. 18: 517-27. PMID 21326331 DOI: 10.1038/Gt.2010.179 |
0.663 |
|
| 2011 |
Bell P, Gao G, Haskins ME, Wang L, Sleeper M, Wang H, Calcedo R, Vandenberghe LH, Chen SJ, Weisse C, Withnall E, Wilson JM. Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. Human Gene Therapy. 22: 985-97. PMID 21204705 DOI: 10.1089/Hum.2010.194 |
0.548 |
|
| 2010 |
Kassim SH, Li H, Vandenberghe LH, Hinderer C, Bell P, Marchadier D, Wilson A, Cromley D, Redon V, Yu H, Wilson JM, Rader DJ. Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis. Plos One. 5: e13424. PMID 20976059 DOI: 10.1371/Journal.Pone.0013424 |
0.798 |
|
| 2010 |
Carlon M, Toelen J, Van der Perren A, Vandenberghe LH, Reumers V, Sbragia L, Gijsbers R, Baekelandt V, Himmelreich U, Wilson JM, Deprest J, Debyser Z. Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 2130-8. PMID 20664525 DOI: 10.1038/Mt.2010.153 |
0.661 |
|
| 2010 |
Vandenberghe LH, Xiao R, Lock M, Lin J, Korn M, Wilson JM. Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. Human Gene Therapy. 21: 1251-7. PMID 20649475 DOI: 10.1089/Hum.2010.107 |
0.548 |
|
| 2010 |
Lock M, Alvira M, Vandenberghe LH, Samanta A, Toelen J, Debyser Z, Wilson JM. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Human Gene Therapy. 21: 1259-71. PMID 20497038 DOI: 10.1089/Hum.2010.055 |
0.652 |
|
| 2010 |
Wang L, Calcedo R, Wang H, Bell P, Grant R, Vandenberghe LH, Sanmiguel J, Morizono H, Batshaw ML, Wilson JM. The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 126-34. PMID 19888196 DOI: 10.1038/Mt.2009.245 |
0.578 |
|
| 2010 |
Wang L, Wang H, Bell P, McCarter RJ, He J, Calcedo R, Vandenberghe LH, Morizono H, Batshaw ML, Wilson JM. Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 118-25. PMID 19861950 DOI: 10.1038/Mt.2009.246 |
0.574 |
|
| 2009 |
Lin J, Calcedo R, Vandenberghe LH, Bell P, Somanathan S, Wilson JM. A new genetic vaccine platform based on an adeno-associated virus isolated from a rhesus macaque. Journal of Virology. 83: 12738-50. PMID 19812149 DOI: 10.1128/Jvi.01441-09 |
0.528 |
|
| 2009 |
Vandenberghe LH, Breous E, Nam HJ, Gao G, Xiao R, Sandhu A, Johnston J, Debyser Z, Agbandje-McKenna M, Wilson JM. Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Therapy. 16: 1416-28. PMID 19727141 DOI: 10.1038/Gt.2009.101 |
0.658 |
|
| 2009 |
Roy S, Vandenberghe LH, Kryazhimskiy S, Grant R, Calcedo R, Yuan X, Keough M, Sandhu A, Wang Q, Medina-Jaszek CA, Plotkin JB, Wilson JM. Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates. Plos Pathogens. 5: e1000503. PMID 19578438 DOI: 10.1371/Journal.Ppat.1000503 |
0.475 |
|
| 2009 |
Breous E, Somanathan S, Vandenberghe LH, Wilson JM. Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver. Hepatology (Baltimore, Md.). 50: 612-21. PMID 19575456 DOI: 10.1002/Hep.23043 |
0.44 |
|
| 2009 |
Gao G, Wang Q, Calcedo R, Mays L, Bell P, Wang L, Vandenberghe LH, Grant R, Sanmiguel J, Furth EE, Wilson JM. Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Human Gene Therapy. 20: 930-42. PMID 19441963 DOI: 10.1089/Hum.2009.060 |
0.757 |
|
| 2009 |
Mays LE, Vandenberghe LH, Xiao R, Bell P, Nam HJ, Agbandje-McKenna M, Wilson JM. Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins. Journal of Immunology (Baltimore, Md. : 1950). 182: 6051-60. PMID 19414756 DOI: 10.4049/Jimmunol.0803965 |
0.718 |
|
| 2009 |
Song Y, Lou HH, Boyer JL, Limberis MP, Vandenberghe LH, Hackett NR, Leopold PL, Wilson JM, Crystal RG. Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Human Gene Therapy. 20: 267-81. PMID 19257851 DOI: 10.1089/Hum.2008.173 |
0.457 |
|
| 2009 |
Calcedo R, Vandenberghe LH, Gao G, Lin J, Wilson JM. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. The Journal of Infectious Diseases. 199: 381-90. PMID 19133809 DOI: 10.1086/595830 |
0.495 |
|
| 2009 |
Calcedo R, Vandenberghe LH, Roy S, Somanathan S, Wang L, Wilson JM. Host immune responses to chronic adenovirus infections in human and nonhuman primates. Journal of Virology. 83: 2623-31. PMID 19116257 DOI: 10.1128/Jvi.02160-08 |
0.495 |
|
| 2009 |
Limberis MP, Vandenberghe LH, Zhang L, Pickles RJ, Wilson JM. Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 294-301. PMID 19066597 DOI: 10.1038/Mt.2008.261 |
0.54 |
|
| 2009 |
Vandenberghe LH, Wilson JM, Gao G. Tailoring the AAV vector capsid for gene therapy. Gene Therapy. 16: 311-9. PMID 19052631 DOI: 10.1038/Gt.2008.170 |
0.584 |
|
| 2009 |
Song Y, Lou H, Boyer J, Limberis M, Vandenberghe L, Hackett N, Leopold P, Wilson J, Crystal R. Functional CFTR Expression in Cystic Fibrosis Airway Epithelial Cells by AAV6.2-mediated Segmental Trans-splicing Human Gene Therapy. 90205172040003. DOI: 10.1089/Hgt.2008.173 |
0.416 |
|
| 2008 |
Cearley CN, Vandenberghe LH, Parente MK, Carnish ER, Wilson JM, Wolfe JH. Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1710-8. PMID 18714307 DOI: 10.1038/Mt.2008.166 |
0.535 |
|
| 2008 |
Lin J, Calcedo R, Vandenberghe LH, Figueredo JM, Wilson JM. Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines. Human Gene Therapy. 19: 663-9. PMID 18549307 DOI: 10.1089/Hum.2008.033 |
0.514 |
|
| 2008 |
Royo NC, Vandenberghe LH, Ma JY, Hauspurg A, Yu L, Maronski M, Johnston J, Dichter MA, Wilson JM, Watson DJ. Specific AAV serotypes stably transduce primary hippocampal and cortical cultures with high efficiency and low toxicity. Brain Research. 1190: 15-22. PMID 18054899 DOI: 10.1016/J.Brainres.2007.11.015 |
0.472 |
|
| 2007 |
Vandenberghe LH, Wilson JM. AAV as an immunogen. Current Gene Therapy. 7: 325-33. PMID 17979679 DOI: 10.2174/156652307782151416 |
0.583 |
|
| 2007 |
Allocca M, Mussolino C, Garcia-Hoyos M, Sanges D, Iodice C, Petrillo M, Vandenberghe LH, Wilson JM, Marigo V, Surace EM, Auricchio A. Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. Journal of Virology. 81: 11372-80. PMID 17699581 DOI: 10.1128/Jvi.01327-07 |
0.794 |
|
| 2007 |
Bell P, Vandenberghe LH, Wu D, Johnston J, Limberis M, Wilson JM. A comparative analysis of novel fluorescent proteins as reporters for gene transfer studies. The Journal of Histochemistry and Cytochemistry : Official Journal of the Histochemistry Society. 55: 931-9. PMID 17510373 DOI: 10.1369/Jhc.7A7180.2007 |
0.52 |
|
| 2007 |
Taymans JM, Vandenberghe LH, Haute CV, Thiry I, Deroose CM, Mortelmans L, Wilson JM, Debyser Z, Baekelandt V. Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Human Gene Therapy. 18: 195-206. PMID 17343566 DOI: 10.1089/Hum.2006.178 |
0.655 |
|
| 2006 |
Vandenberghe LH, Wang L, Somanathan S, Zhi Y, Figueredo J, Calcedo R, Sanmiguel J, Desai RA, Chen CS, Johnston J, Grant RL, Gao G, Wilson JM. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nature Medicine. 12: 967-71. PMID 16845388 DOI: 10.1038/Nm1445 |
0.526 |
|
| 2006 |
Royo NC, Vandenberghe LH, Johnston J, Maronski M, Dichter MA, Wilson JM, Watson DJ. 900. Specific AAV Serotypes Stably Transduce Hippocampal and Cortical Cultures with High Efficiency and Low Toxicity Molecular Therapy. 13: S347. DOI: 10.1016/J.Ymthe.2006.08.989 |
0.468 |
|
| 2006 |
Limberis M, Vandenberghe LH, Johnston JC, Wu D, McMenamin D, Munden RL, Wilson JM. 684. Improved Performance of AAV6 in Airways In Vivo Molecular Therapy. 13: S264-S265. DOI: 10.1016/J.Ymthe.2006.08.762 |
0.517 |
|
| 2006 |
Vandenberghe LH, Miller JR, Gao G, Calcedo R, Wilson JM. 116. The Proposed AAV Serotypes 10 and 11 Serologically Cross-React with AAV8 and AAV4 Molecular Therapy. 13: S47-S48. DOI: 10.1016/J.Ymthe.2006.08.136 |
0.566 |
|
| 2005 |
Gao G, Vandenberghe LH, Wilson JM. New recombinant serotypes of AAV vectors. Current Gene Therapy. 5: 285-97. PMID 15975006 DOI: 10.2174/1566523054065057 |
0.592 |
|
| 2005 |
Vandenberghe LH, Yang MH, Johnston J, Gao G, Wilson JM. 503. Mutational Strategy To Enhance or Rescue Functionality of Existing AAV Vectors Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.043 |
0.566 |
|
| 2004 |
Roy S, Gao G, Clawson DS, Vandenberghe LH, Farina SF, Wilson JM. Complete nucleotide sequences and genome organization of four chimpanzee adenoviruses. Virology. 324: 361-72. PMID 15207622 DOI: 10.1016/J.Virol.2004.03.047 |
0.446 |
|
| 2004 |
Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, Wilson JM. Clades of Adeno-associated viruses are widely disseminated in human tissues. Journal of Virology. 78: 6381-8. PMID 15163731 DOI: 10.1128/Jvi.78.12.6381-6388.2004 |
0.53 |
|
| 2004 |
Vandenberghe LH, Gao G, Alvira MR, Calcedo R, Wilson JM. 88. AAV clades: Their Ability To Recombine and Cross Species-Barriers Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.024 |
0.519 |
|
| 2003 |
Simmons G, Rennekamp AJ, Chai N, Vandenberghe LH, Riley JL, Bates P. Folate receptor alpha and caveolae are not required for Ebola virus glycoprotein-mediated viral infection. Journal of Virology. 77: 13433-8. PMID 14645601 DOI: 10.1128/Jvi.77.24.13433-13438.2003 |
0.345 |
|
| 2003 |
Parry RV, Rumbley CA, Vandenberghe LH, June CH, Riley JL. CD28 and inducible costimulatory protein Src homology 2 binding domains show distinct regulation of phosphatidylinositol 3-kinase, Bcl-xL, and IL-2 expression in primary human CD4 T lymphocytes. Journal of Immunology (Baltimore, Md. : 1950). 171: 166-74. PMID 12816995 DOI: 10.4049/Jimmunol.171.1.166 |
0.407 |
|
| 2003 |
Gao G, Alvira MR, Somanathan S, Lu Y, Vandenberghe LH, Rux JJ, Calcedo R, Sanmiguel J, Abbas Z, Wilson JM. Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proceedings of the National Academy of Sciences of the United States of America. 100: 6081-6. PMID 12716974 DOI: 10.1073/Pnas.0937739100 |
0.508 |
|
| 2003 |
Simmons G, Reeves JD, Grogan CC, Vandenberghe LH, Baribaud F, Whitbeck JC, Burke E, Buchmeier MJ, Soilleux EJ, Riley JL, Doms RW, Bates P, Pöhlmann S. DC-SIGN and DC-SIGNR bind ebola glycoproteins and enhance infection of macrophages and endothelial cells. Virology. 305: 115-23. PMID 12504546 DOI: 10.1006/Viro.2002.1730 |
0.346 |
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