Year |
Citation |
Score |
2024 |
Suchy FP, Karigane D, Nakauchi Y, Higuchi M, Zhang J, Pekrun K, Hsu I, Fan AC, Nishimura T, Charlesworth CT, Bhadury J, Nishimura T, Wilkinson AC, Kay MA, Majeti R, et al. Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors. Nature Biotechnology. PMID 38589662 DOI: 10.1038/s41587-024-02171-w |
0.32 |
|
2023 |
Gonzalez-Sandoval A, Pekrun K, Tsuji S, Zhang F, Hung KL, Chang HY, Kay MA. The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner. Nature Communications. 14: 2448. PMID 37117181 DOI: 10.1038/s41467-023-38106-3 |
0.349 |
|
2023 |
Puzzo F, Zhang C, Powell Gray B, Zhang F, Sullenger BA, Kay MA. Aptamer-programmable adeno-associated viral vectors as a novel platform for cell-specific gene transfer. Molecular Therapy. Nucleic Acids. 31: 383-397. PMID 36817723 DOI: 10.1016/j.omtn.2023.01.007 |
0.379 |
|
2022 |
Sabatino DE, Bushman CFD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J. Evaluating the State of the Science for Adeno-Associated Virus (AAV) Integration:An Integrated Perspective. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 35690906 DOI: 10.1016/j.ymthe.2022.06.004 |
0.353 |
|
2022 |
Tsuji S, Stephens CJ, Bortolussi G, Zhang F, Baj G, Jang H, de Alencastro G, Muro AF, Pekrun K, Kay MA. Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice. Nature Biotechnology. PMID 35393561 DOI: 10.1038/s41587-022-01240-2 |
0.369 |
|
2022 |
Lisjak M, De Caneva A, Marais T, Barbon E, Biferi MG, Porro F, Barzel A, Zentilin L, Kay MA, Mingozzi F, Muro AF. Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice. Frontiers in Genome Editing. 4: 785698. PMID 35359664 DOI: 10.3389/fgeed.2022.785698 |
0.371 |
|
2020 |
Chandler RJ, Venturoni LE, Liao J, Hubbard BT, Schneller JL, Hoffmann V, Gordo S, Zang S, Ko CW, Chau N, Chiang K, Kay MA, Barzel A, Venditti CP. Promoterless, nuclease-free genome editing confers a growth advantage for corrected hepatocytes in mice with methylmalonic acidemia. Hepatology (Baltimore, Md.). PMID 32976669 DOI: 10.1002/hep.31570 |
0.369 |
|
2020 |
Kim HK, Yeom JH, Kay MA. Transfer RNA-Derived Small RNAs: Another Layer of Gene Regulation and Novel Targets for Disease Therapeutics. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 32956625 DOI: 10.1016/J.Ymthe.2020.09.013 |
0.397 |
|
2020 |
Course MM, Gudsnuk K, Smukowski SN, Winston K, Desai N, Ross JP, Sulovari A, Bourassa CV, Spiegelman D, Couthouis J, Yu CE, Tsuang DW, Jayadev S, Kay MA, Gitler AD, et al. Evolution of a Human-Specific Tandem Repeat Associated with ALS. American Journal of Human Genetics. PMID 32750315 DOI: 10.1016/J.Ajhg.2020.07.004 |
0.333 |
|
2020 |
Su Y, Walker JR, Park Y, Smith TP, Liu LX, Hall MP, Labanieh L, Hurst R, Wang DC, Encell LP, Kim N, Zhang F, Kay MA, Casey KM, Majzner RG, et al. Novel NanoLuc substrates enable bright two-population bioluminescence imaging in animals. Nature Methods. PMID 32661427 DOI: 10.1038/S41592-020-0889-6 |
0.316 |
|
2020 |
de Alencastro G, Pekrun K, Valdmanis P, Tiffany M, Xu J, Kay MA. Tracking adeno-associated virus capsid evolution by high-throughput sequencing. Human Gene Therapy. PMID 32024384 DOI: 10.1089/Hum.2019.339 |
0.404 |
|
2020 |
Kim HK, Xu J, Chu K, Park H, jang H, Li P, Valdmanis P, Zhang Q, Kay M. Abstract LB-343: A Leu(CAG)-tRNA derived small RNA regulates ribosomal protein S28 after translation initiation in both human and mouse liver cancers Cancer Research. 80. DOI: 10.1158/1538-7445.Am2020-Lb-343 |
0.373 |
|
2020 |
Kay MA. The Role of tRNA Derived Small RNAs in Gene Regulation in Normal Tissues and Cancer The Faseb Journal. 34: 1-1. DOI: 10.1096/Fasebj.2020.34.S1.00158 |
0.335 |
|
2019 |
Kim HK, Xu J, Chu K, Park H, Jang H, Li P, Valdmanis PN, Zhang QC, Kay MA. A tRNA-Derived Small RNA Regulates Ribosomal Protein S28 Protein Levels after Translation Initiation in Humans and Mice. Cell Reports. 29: 3816-3824.e4. PMID 31851915 DOI: 10.1016/J.Celrep.2019.11.062 |
0.347 |
|
2019 |
Pekrun K, De Alencastro G, Luo QJ, Liu J, Kim Y, Nygaard S, Galivo F, Zhang F, Song R, Tiffany MR, Xu J, Hebrok M, Grompe M, Kay MA. Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors. Jci Insight. 4. PMID 31723052 DOI: 10.1172/Jci.Insight.131610 |
0.447 |
|
2019 |
Zaleta-Rivera K, Dainis A, Ribeiro AJS, Sanchez Cordero P, Rubio G, Shang C, Liu J, Finsterbach T, Parikh VN, Sutton S, Seo K, Sinha N, Jain N, Huang Y, Hajjar RJ, ... Kay MA, et al. Allele-Specific Silencing Ameliorates Restrictive Cardiomyopathy Due to a Human Myosin Regulatory Light Chain Mutation. Circulation. PMID 31315475 DOI: 10.1161/Circulationaha.118.036965 |
0.319 |
|
2019 |
De Caneva A, Porro F, Bortolussi G, Sola R, Lisjak M, Barzel A, Giacca M, Kay MA, Vlahoviček K, Zentilin L, Muro AF. Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases. Jci Insight. 5. PMID 31211694 DOI: 10.1172/Jci.Insight.128863 |
0.475 |
|
2018 |
Valdmanis PN, Kim HK, Chu K, Zhang F, Xu J, Munding EM, Shen J, Kay MA. miR-122 removal in the liver activates imprinted microRNAs and enables more effective microRNA-mediated gene repression. Nature Communications. 9: 5321. PMID 30552326 DOI: 10.1038/S41467-018-07786-7 |
0.324 |
|
2018 |
Kim HK, Fuchs G, Wang S, Wei W, Zhang Y, Park H, Roy-Chaudhuri B, Li P, Xu J, Chu K, Zhang F, Chua M, So S, Zhang C, Sarnow P, ... Kay MA, et al. Abstract LB-390: A transfer RNA derived small RNA affects translation in rapidly dividing cells and a target for hepatocellular carcinoma Cancer Research. 78. DOI: 10.1158/1538-7445.Am2018-Lb-390 |
0.393 |
|
2017 |
Winters IP, Chiou SH, Paulk NK, McFarland CD, Lalgudi PV, Ma RK, Lisowski L, Connolly AJ, Petrov DA, Kay MA, Winslow MM. Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity. Nature Communications. 8: 2053. PMID 29233960 DOI: 10.1038/S41467-017-01519-Y |
0.309 |
|
2017 |
Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, ... ... Kay MA, et al. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. Science Translational Medicine. 9. PMID 29187643 DOI: 10.1126/Scitranslmed.Aam6375 |
0.429 |
|
2017 |
Kim HK, Fuchs G, Wang S, Wei W, Zhang Y, Park H, Roy-Chaudhuri B, Li P, Xu J, Chu K, Zhang F, Chua MS, So S, Zhang QC, Sarnow P, ... Kay MA, et al. A transfer-RNA-derived small RNA regulates ribosome biogenesis. Nature. 552: 57-62. PMID 29186115 DOI: 10.1038/Nature25005 |
0.357 |
|
2017 |
Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, ... ... Kay MA, et al. Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 29055620 DOI: 10.1016/J.Ymthe.2017.09.021 |
0.423 |
|
2017 |
Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C. Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 29032169 DOI: 10.1016/J.Ymthe.2017.09.020 |
0.45 |
|
2017 |
Porro F, Bortolussi G, Barzel A, De Caneva A, Iaconcig A, Vodret S, Zentilin L, Kay MA, Muro AF. Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model. Embo Molecular Medicine. PMID 28751579 DOI: 10.15252/Emmm.201707601 |
0.438 |
|
2017 |
Lu J, Zhang F, Fire AZ, Kay MA. Sequence-Modified Antibiotic Resistance Genes Provide Sustained Plasmid-Mediated Transgene Expression in Mammals. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28365028 DOI: 10.1016/J.Ymthe.2017.03.003 |
0.467 |
|
2017 |
Valdmanis P, Kay MA. Future of rAAV gene therapy: Platform for RNAi, Gene Editing and Beyond. Human Gene Therapy. PMID 28073291 DOI: 10.1089/Hum.2016.171 |
0.439 |
|
2017 |
Colella P, Puzzo F, Biferi M, Bali D, Paulk N, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Sellier P, Wittenberghe Lv, Boisgerault F, Barkats M, ... ... Kay M, et al. Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.543 |
0.332 |
|
2016 |
Wang Y, Pryputniewicz-Dobrinska D, Nagy EÉ, Kaufman CD, Singh M, Yant S, Wang J, Dalda A, Kay MA, Ivics Z, Izsvák Z. Regulated complex assembly safeguards the fidelity of Sleeping Beauty transposition. Nucleic Acids Research. PMID 27913727 DOI: 10.1093/Nar/Gkw1164 |
0.35 |
|
2016 |
Lu J, Williams JA, Luke J, Zhang F, Chu K, Kay MA. A 5'non-coding exon containing engineered intron enhances transgene expression from recombinant AAV vectors in vivo. Human Gene Therapy. PMID 27903072 DOI: 10.1089/hum.2016.140 |
0.366 |
|
2016 |
Nygaard S, Barzel A, Haft A, Major A, Finegold M, Kay MA, Grompe M. A universal system to select gene-modified hepatocytes in vivo. Science Translational Medicine. 8: 342ra79. PMID 27280686 DOI: 10.1126/Scitranslmed.Aad8166 |
0.487 |
|
2016 |
Valdmanis PN, Gu S, Chu K, Jin L, Zhang F, Munding EM, Zhang Y, Huang Y, Kutay H, Ghoshal K, Lisowski L, Kay MA. RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice. Nature Medicine. PMID 27064447 DOI: 10.1038/Nm.4079 |
0.409 |
|
2016 |
Valdmanis PN, Gu S, Chu K, Jin L, Zhang F, Munding EM, Zhang Y, Huang Y, Kutay H, Ghoshal K, Lisowski L, Kay MA. 737. RNAi Induced Hepatotoxicity Results from a Functional Depletion of the First Synthesized Isoform of miR-122 Molecular Therapy. 24: S290-S291. DOI: 10.1016/S1525-0016(16)33545-6 |
0.307 |
|
2016 |
de Alencastro G, Pekrun K, Kay M. 539. Screening for Recombinant Adeno-Associated Viral Vectors That Selectively Transduce Hepatitis B Virus Infected Cells Molecular Therapy. 24: S215. DOI: 10.1016/S1525-0016(16)33347-0 |
0.406 |
|
2016 |
Muench R, Kay M. 257. Selection of Next Generation AAV Gene Therapy Vectors for Specific and Precise Gene Delivery Molecular Therapy. 24: S101. DOI: 10.1016/S1525-0016(16)33066-0 |
0.447 |
|
2016 |
Tiffany M, Kay MA. 253. Expanded Packaging Capacity of AAV by Lumenal Charge Alteration Molecular Therapy. 24: S99-S100. DOI: 10.1016/S1525-0016(16)33062-3 |
0.464 |
|
2016 |
Spector LP, Kay MA. 129. Does Transcription Influence AAV-Mediated Homologous Recombination? Molecular Therapy. 24: S53. DOI: 10.1016/S1525-0016(16)32938-0 |
0.521 |
|
2016 |
Chandler RJ, Barzel A, Kay MA, Venditti CP. 48. Treatment of Methylmalonic Acidemia by Promoterless Gene-Targeting Using Adeno-Associated Viral (AAV) Mediated Homologous Recombination Molecular Therapy. 24: S21-S22. DOI: 10.1016/S1525-0016(16)32857-X |
0.486 |
|
2015 |
Mellins ED, Kay MA. Viral Vectors Take On HIV Infection. The New England Journal of Medicine. 373: 770-2. PMID 26287853 DOI: 10.1056/Nejmcibr1504232 |
0.334 |
|
2015 |
Nichols TC, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA. Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Human Gene Therapy. Clinical Development. 26: 5-14. PMID 25675273 DOI: 10.1089/Humc.2014.153 |
0.444 |
|
2015 |
Diecke S, Lu J, Lee J, Termglinchan V, Kooreman NG, Burridge PW, Ebert AD, Churko JM, Sharma A, Kay MA, Wu JC. Novel codon-optimized mini-intronic plasmid for efficient, inexpensive, and xeno-free induction of pluripotency. Scientific Reports. 5: 8081. PMID 25628230 DOI: 10.1038/Srep08081 |
0.442 |
|
2015 |
Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature. 517: 360-4. PMID 25363772 DOI: 10.1038/Nature13864 |
0.476 |
|
2015 |
Porro F, Bortolussi G, Barzel A, Zentilin L, Vodret S, Bockor L, Kay MA, Muro AF. 688. AAV8-Mediated Liver Gene Targeting Without Nucleases Rescues Lethality in a Mouse Model of the Crigler-Najjar Syndrome Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)34297-6 |
0.505 |
|
2015 |
Nygaard S, Barzel A, Haft A, Kay MA, Grompe M. 683. In Vivo Expansion of Hepatocytes with Targeted rAAV Integration Results in a >100-Fold Increase of Transgene Expression Molecular Therapy. 23: S272. DOI: 10.1016/S1525-0016(16)34292-7 |
0.482 |
|
2015 |
de Alencastro G, Paulk NK, Pekrun K, Kay MA. 305. A Screening Strategy for Selecting Recombinant Adeno-Associated Viral Vectors That Selectively Transduce Viral Infected Cells Molecular Therapy. 23: S123. DOI: 10.1016/S1525-0016(16)33914-4 |
0.423 |
|
2015 |
Paulk NK, Charville GW, Pekrun K, Maguire K, Rando TA, Kay MA. 303. AAV Capsid Evolution for Enhanced Antibody Delivery To Human Muscle for Use in Next-Generation HIV Vaccines Molecular Therapy. 23: S122-S123. DOI: 10.1016/S1525-0016(16)33912-0 |
0.345 |
|
2015 |
Paulk NK, Pekrun K, Lisowski L, Zhang Y, Chu K, Kay MA. 262. Directed Evolution of Improved AAV Capsids for the Ideal Human Liver Vector–Can Human Liver Tropism and Human Immune Evasion Be Achieved? Molecular Therapy. 23: S104-S105. DOI: 10.1016/S1525-0016(16)33871-0 |
0.415 |
|
2015 |
Tiffany MR, Kay MA. 94. AAV Integration Site Determination Using Illumina Mate Pair Sequencing Molecular Therapy. 23: S39-S40. DOI: 10.1016/S1525-0016(16)33699-1 |
0.349 |
|
2014 |
Nichols T, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA. Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs. Human Gene Therapy. Clinical Development. PMID 25548971 DOI: 10.1089/Hum.2014.153 |
0.413 |
|
2014 |
Sebastiano V, Zhen HH, Haddad B, Derafshi BH, Bashkirova E, Melo SP, Wang P, Leung TL, Siprashvili Z, Tichy A, Li J, Ameen M, Hawkins J, Lee S, Li L, ... ... Kay MA, et al. Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa. Science Translational Medicine. 6: 264ra163. PMID 25429056 DOI: 10.1126/Scitranslmed.3009540 |
0.352 |
|
2014 |
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, ... ... Kay MA, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. The New England Journal of Medicine. 371: 1994-2004. PMID 25409372 DOI: 10.1056/Nejmoa1407309 |
0.416 |
|
2014 |
Gu S, Zhang Y, Jin L, Huang Y, Zhang F, Bassik MC, Kampmann M, Kay MA. Weak base pairing in both seed and 3' regions reduces RNAi off-targets and enhances si/shRNA designs. Nucleic Acids Research. 42: 12169-76. PMID 25270879 DOI: 10.1093/Nar/Gku854 |
0.363 |
|
2014 |
Ehmer U, Zmoos AF, Auerbach RK, Vaka D, Butte AJ, Kay MA, Sage J. Organ size control is dominant over Rb family inactivation to restrict proliferation in vivo. Cell Reports. 8: 371-81. PMID 25017070 DOI: 10.1016/J.Celrep.2014.06.025 |
0.331 |
|
2014 |
Phillips N, Kay MA. Characterization of vector-based delivery of neurogenin-3 in murine diabetes. Human Gene Therapy. 25: 651-61. PMID 24635696 DOI: 10.1089/Hum.2013.206 |
0.311 |
|
2014 |
Lisowski L, Dane AP, Chu K, Zhang Y, Cunningham SC, Wilson EM, Nygaard S, Grompe M, Alexander IE, Kay MA. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature. 506: 382-6. PMID 24390344 DOI: 10.1038/Nature12875 |
0.431 |
|
2014 |
Melo SP, Lisowski L, Bashkirova E, Zhen HH, Chu K, Keene DR, Marinkovich MP, Kay MA, Oro AE. Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 725-33. PMID 24390279 DOI: 10.1038/Mt.2013.290 |
0.434 |
|
2014 |
Borel F, Kay MA, Mueller C. Recombinant AAV as a platform for translating the therapeutic potential of RNA interference. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 692-701. PMID 24352214 DOI: 10.1038/Mt.2013.285 |
0.401 |
|
2013 |
Valdmanis PN, Kay MA. The expanding repertoire of circular RNAs. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1112-4. PMID 23728253 DOI: 10.1038/Mt.2013.101 |
0.343 |
|
2013 |
Lu J, Zhang F, Kay MA. A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 954-63. PMID 23459514 DOI: 10.1038/Mt.2013.33 |
0.502 |
|
2013 |
Lisowski L, Elazar M, Chu K, Glenn JS, Kay MA. The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA. Nucleic Acids Research. 41: 3688-98. PMID 23396439 DOI: 10.1093/Nar/Gkt068 |
0.322 |
|
2013 |
Gracey Maniar LE, Maniar JM, Chen ZY, Lu J, Fire AZ, Kay MA. Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 131-8. PMID 23183534 DOI: 10.1038/Mt.2012.244 |
0.457 |
|
2013 |
Basner-Tschakarjan E, Mingozzi F, Chen Y, Nathwani A, Tuddenham E, Riddell A, Ng C, Rustagi P, Glader B, Kay M, Reiss UM, Nienhuis AW, Davidoff A, High KA. Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector Blood. 122: 717-717. DOI: 10.1182/Blood.V122.21.717.717 |
0.35 |
|
2012 |
Valdmanis PN, Lisowski L, Kay MA. rAAV-mediated tumorigenesis: still unresolved after an AAV assault. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 2014-7. PMID 23131853 DOI: 10.1038/Mt.2012.220 |
0.419 |
|
2012 |
Wang Z, Lisowski L, Finegold MJ, Nakai H, Kay MA, Grompe M. AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1902-11. PMID 22990673 DOI: 10.1038/Mt.2012.157 |
0.492 |
|
2012 |
Lisowski L, Lau A, Wang Z, Zhang Y, Zhang F, Grompe M, Kay MA. Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1912-23. PMID 22990671 DOI: 10.1038/Mt.2012.164 |
0.527 |
|
2012 |
Wang Y, Zhang WY, Hu S, Lan F, Lee AS, Huber B, Lisowski L, Liang P, Huang M, de Almeida PE, Won JH, Sun N, Robbins RC, Kay MA, Urnov FD, et al. Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circulation Research. 111: 1494-503. PMID 22967807 DOI: 10.1161/Circresaha.112.274969 |
0.382 |
|
2012 |
Gu S, Jin L, Huang Y, Zhang F, Kay MA. Slicing-independent RISC activation requires the argonaute PAZ domain. Current Biology : Cb. 22: 1536-42. PMID 22795694 DOI: 10.1016/J.Cub.2012.06.040 |
0.306 |
|
2012 |
Lu J, Zhang F, Xu S, Fire AZ, Kay MA. The extragenic spacer length between the 5' and 3' ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 2111-9. PMID 22565847 DOI: 10.1038/Mt.2012.65 |
0.433 |
|
2012 |
Valdmanis PN, Gu S, Schüermann N, Sethupathy P, Grimm D, Kay MA. Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucleic Acids Research. 40: 3704-13. PMID 22210886 DOI: 10.1093/Nar/Gkr1274 |
0.57 |
|
2012 |
Davidoff A, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Chowdary P, Riddell A, Glader B, Rustagi P, Ng C, Kay M, Zhou J, Spence Y, Morton C, Allay J, et al. Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B Blood. 120: 752-752. DOI: 10.1182/Blood.V120.21.752.752 |
0.44 |
|
2011 |
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, ... ... Kay MA, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. The New England Journal of Medicine. 365: 2357-65. PMID 22149959 DOI: 10.1056/Nejmoa1108046 |
0.455 |
|
2011 |
Malato Y, Naqvi S, Schürmann N, Ng R, Wang B, Zape J, Kay MA, Grimm D, Willenbring H. Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration. The Journal of Clinical Investigation. 121: 4850-60. PMID 22105172 DOI: 10.1172/Jci59261 |
0.58 |
|
2011 |
Huang M, Nguyen P, Jia F, Hu S, Gong Y, de Almeida PE, Wang L, Nag D, Kay MA, Giaccia AJ, Robbins RC, Wu JC. Double knockdown of prolyl hydroxylase and factor-inhibiting hypoxia-inducible factor with nonviral minicircle gene therapy enhances stem cell mobilization and angiogenesis after myocardial infarction. Circulation. 124: S46-54. PMID 21911818 DOI: 10.1161/Circulationaha.110.014019 |
0.36 |
|
2011 |
Deuse T, Seifert M, Phillips N, Fire A, Tyan D, Kay M, Tsao PS, Hua X, Velden J, Eiermann T, Volk HD, Reichenspurner H, Robbins RC, Schrepfer S. Human leukocyte antigen I knockdown human embryonic stem cells induce host ignorance and achieve prolonged xenogeneic survival. Circulation. 124: S3-9. PMID 21911816 DOI: 10.1161/Circulationaha.111.020727 |
0.353 |
|
2011 |
Deuse T, Seifert M, Phillips N, Fire A, Tyan D, Kay M, Tsao PS, Hua X, Velden J, Eiermann T, Volk HD, Reichenspurner H, Robbins RC, Schrepfer S. Immunobiology of naïve and genetically modified HLA-class-I-knockdown human embryonic stem cells. Journal of Cell Science. 124: 3029-37. PMID 21878509 DOI: 10.1242/Jcs.087718 |
0.316 |
|
2011 |
Gu S, Jin L, Zhang F, Huang Y, Grimm D, Rossi JJ, Kay MA. Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes. Proceedings of the National Academy of Sciences of the United States of America. 108: 9208-13. PMID 21576459 DOI: 10.1073/Pnas.1018023108 |
0.535 |
|
2011 |
Kay MA. State-of-the-art gene-based therapies: the road ahead. Nature Reviews. Genetics. 12: 316-28. PMID 21468099 DOI: 10.1038/Nrg2971 |
0.357 |
|
2011 |
Narsinh KH, Jia F, Robbins RC, Kay MA, Longaker MT, Wu JC. Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Nature Protocols. 6: 78-88. PMID 21212777 DOI: 10.1038/Nprot.2010.173 |
0.403 |
|
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 |
0.483 |
|
2011 |
Deuse T, Stubbendorff M, Tang-Quan K, Phillips N, Kay MA, Eiermann T, Phan TT, Volk HD, Reichenspurner H, Robbins RC, Schrepfer S. Immunogenicity and immunomodulatory properties of umbilical cord lining mesenchymal stem cells. Cell Transplantation. 20: 655-67. PMID 21054940 DOI: 10.3727/096368910X536473 |
0.335 |
|
2011 |
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh JH, Linch DC, Chowdary P, Griffioen A, Riddell A, Pie J, Harrington C, O’Beirne J, Smith K, Pasi J, Glader B, ... ... Kay M, et al. Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B Blood. 118: 5-5. DOI: 10.1182/Blood.V118.21.5.5 |
0.44 |
|
2011 |
Deuse T, Seifert M, Fire A, Phillips N, Hua X, Velden J, Kay M, Volk H, Reichenspurner H, Robbins R, Schrepfer S. 547 Hypoimmunogeneic HLA I Knockdown Human Embryonic Stem Cells Induce Host Ignorance and Achieve Prolonged Xenogeneic Survival The Journal of Heart and Lung Transplantation. 30: S184. DOI: 10.1016/J.Healun.2011.01.558 |
0.312 |
|
2010 |
Kay MA, He CY, Chen ZY. A robust system for production of minicircle DNA vectors. Nature Biotechnology. 28: 1287-9. PMID 21102455 DOI: 10.1038/Nbt.1708 |
0.386 |
|
2010 |
Hausl MA, Zhang W, Müther N, Rauschhuber C, Franck HG, Merricks EP, Nichols TC, Kay MA, Ehrhardt A. Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 1896-906. PMID 20717103 DOI: 10.1038/Mt.2010.169 |
0.503 |
|
2010 |
Grimm D, Wang L, Lee JS, Schürmann N, Gu S, Börner K, Storm TA, Kay MA. Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. The Journal of Clinical Investigation. 120: 3106-19. PMID 20697157 DOI: 10.1172/Jci43565 |
0.655 |
|
2010 |
Gracey LE, Chen ZY, Maniar JM, Valouev A, Sidow A, Kay MA, Fire AZ. An in vitro-identified high-affinity nucleosome-positioning signal is capable of transiently positioning a nucleosome in vivo. Epigenetics & Chromatin. 3: 13. PMID 20594331 DOI: 10.1186/1756-8935-3-13 |
0.331 |
|
2010 |
Nichols TC, Raymer RA, Franck HW, Merricks EP, Bellinger DA, DeFriess N, Margaritis P, Arruda VR, Kay MA, High KA. Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B. Haemophilia : the Official Journal of the World Federation of Hemophilia. 16: 19-23. PMID 20586797 DOI: 10.1111/J.1365-2516.2010.02255.X |
0.348 |
|
2010 |
Falcon A, Doege H, Fluitt A, Tsang B, Watson N, Kay MA, Stahl A. FATP2 is a hepatic fatty acid transporter and peroxisomal very long-chain acyl-CoA synthetase. American Journal of Physiology. Endocrinology and Metabolism. 299: E384-93. PMID 20530735 DOI: 10.1152/Ajpendo.00226.2010 |
0.353 |
|
2010 |
Gu S, Kay MA. How do miRNAs mediate translational repression? Silence. 1: 11. PMID 20459656 DOI: 10.1186/1758-907X-1-11 |
0.306 |
|
2010 |
Haussecker D, Huang Y, Lau A, Parameswaran P, Fire AZ, Kay MA. Human tRNA-derived small RNAs in the global regulation of RNA silencing. Rna (New York, N.Y.). 16: 673-95. PMID 20181738 DOI: 10.1261/Rna.2000810 |
0.369 |
|
2010 |
Parameswaran P, Sklan E, Wilkins C, Burgon T, Samuel MA, Lu R, Ansel KM, Heissmeyer V, Einav S, Jackson W, Doukas T, Paranjape S, Polacek C, dos Santos FB, Jalili R, ... ... Kay M, et al. Six RNA viruses and forty-one hosts: viral small RNAs and modulation of small RNA repertoires in vertebrate and invertebrate systems. Plos Pathogens. 6: e1000764. PMID 20169186 DOI: 10.1371/Journal.Ppat.1000764 |
0.544 |
|
2010 |
Paulk NK, Wursthorn K, Wang Z, Finegold MJ, Kay MA, Grompe M. Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology (Baltimore, Md.). 51: 1200-8. PMID 20162619 DOI: 10.1002/Hep.23481 |
0.465 |
|
2010 |
Jia F, Wilson KD, Sun N, Gupta DM, Huang M, Li Z, Panetta NJ, Chen ZY, Robbins RC, Kay MA, Longaker MT, Wu JC. A nonviral minicircle vector for deriving human iPS cells. Nature Methods. 7: 197-9. PMID 20139967 DOI: 10.1038/Nmeth.1426 |
0.388 |
|
2010 |
Haussecker D, Kay MA. miR-122 continues to blaze the trail for microRNA therapeutics. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 240-2. PMID 20125164 DOI: 10.1038/Mt.2009.313 |
0.335 |
|
2010 |
Beer S, Bellovin DI, Lee JS, Komatsubara K, Wang LS, Koh H, Börner K, Storm TA, Davis CR, Kay MA, Felsher DW, Grimm D. Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 161-70. PMID 19844192 DOI: 10.1038/Mt.2009.222 |
0.617 |
|
2010 |
Nathwani A, Tuddenham E, Rosales C, McIntosh J, Riddell A, Rustagi P, Glader B, Kay M, Allay J, Coleman J, Sleep S, High KA, Mingozzi F, Gray JT, Reiss UM, et al. Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B Blood. 116: 248-248. DOI: 10.1182/Blood.V116.21.248.248 |
0.362 |
|
2009 |
Cao D, Haussecker D, Huang Y, Kay MA. Combined proteomic-RNAi screen for host factors involved in human hepatitis delta virus replication. Rna (New York, N.Y.). 15: 1971-9. PMID 19776158 DOI: 10.1261/Rna.1782209 |
0.392 |
|
2009 |
Huang M, Chen Z, Hu S, Jia F, Li Z, Hoyt G, Robbins RC, Kay MA, Wu JC. Novel minicircle vector for gene therapy in murine myocardial infarction. Circulation. 120: S230-7. PMID 19752373 DOI: 10.1161/Circulationaha.108.841155 |
0.459 |
|
2009 |
Jager L, Hausl MA, Rauschhuber C, Wolf NM, Kay MA, Ehrhardt A. A rapid protocol for construction and production of high-capacity adenoviral vectors. Nature Protocols. 4: 547-64. PMID 19373227 DOI: 10.1038/Nprot.2009.4 |
0.415 |
|
2009 |
Stenler S, Andersson A, Simonson OE, Lundin KE, Chen ZY, Kay MA, Smith CI, Sylvén C, Blomberg P. Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor. Journal of Cardiovascular Pharmacology. 53: 18-23. PMID 19129741 DOI: 10.1097/Fjc.0B013E318194234E |
0.4 |
|
2008 |
Zhang X, Epperly MW, Kay MA, Chen ZY, Dixon T, Franicola D, Greenberger BA, Komanduri P, Greenberger JS. Radioprotection in vitro and in vivo by minicircle plasmid carrying the human manganese superoxide dismutase transgene. Human Gene Therapy. 19: 820-6. PMID 18699723 DOI: 10.1089/Hum.2007.141 |
0.372 |
|
2008 |
Giering JC, Grimm D, Storm TA, Kay MA. Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1630-6. PMID 18665161 DOI: 10.1038/Mt.2008.144 |
0.814 |
|
2008 |
Haussecker D, Cao D, Huang Y, Parameswaran P, Fire AZ, Kay MA. Capped small RNAs and MOV10 in human hepatitis delta virus replication. Nature Structural & Molecular Biology. 15: 714-21. PMID 18552826 DOI: 10.1038/Nsmb.1440 |
0.361 |
|
2008 |
Doege H, Grimm D, Falcon A, Tsang B, Storm TA, Xu H, Ortegon AM, Kazantzis M, Kay MA, Stahl A. Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia. The Journal of Biological Chemistry. 283: 22186-92. PMID 18524776 DOI: 10.1074/Jbc.M803510200 |
0.528 |
|
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 |
0.323 |
|
2008 |
Grimm D, Lee JS, Wang L, Desai T, Akache B, Storm TA, Kay MA. In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. Journal of Virology. 82: 5887-911. PMID 18400866 DOI: 10.1128/Jvi.00254-08 |
0.638 |
|
2008 |
McCaffrey AP, Fawcett P, Nakai H, McCaffrey RL, Ehrhardt A, Pham TT, Pandey K, Xu H, Feuss S, Storm TA, Kay MA. The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 931-41. PMID 18388926 DOI: 10.1038/Mt.2008.37 |
0.494 |
|
2008 |
Chen ZY, Riu E, He CY, Xu H, Kay MA. Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 548-56. PMID 18253155 DOI: 10.1038/Sj.Mt.6300399 |
0.407 |
|
2008 |
Streetz KL, Doyonnas R, Grimm D, Jenkins DD, Fuess S, Perryman S, Lin J, Trautwein C, Shizuru J, Blau H, Sylvester KG, Kay MA. Hepatic parenchymal replacement in mice by transplanted allogeneic hepatocytes is facilitated by bone marrow transplantation and mediated by CD4 cells. Hepatology (Baltimore, Md.). 47: 706-18. PMID 18220289 DOI: 10.1002/Hep.22012 |
0.54 |
|
2008 |
Zhang X, Epperly MW, Kay MA, Chen Z, Smith T, Franicola D, Greenberger BA, Komanduri P, Greenberger JS. Radioprotection In Vitro and In Vivo by Mini Circle Plasmid Containing the Human Manganese Superoxide Dismutase (MnSOD) Transgene Human Gene Therapy. 19: 81015093227032. DOI: 10.1089/Hgt.2007.141 |
0.343 |
|
2007 |
Grimm D, Kay MA. Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? The Journal of Clinical Investigation. 117: 3633-41. PMID 18060021 DOI: 10.1172/Jci34129 |
0.547 |
|
2007 |
Grimm D, Kay MA. RNAi and gene therapy: a mutual attraction. Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program. 473-81. PMID 18024667 DOI: 10.1182/Asheducation-2007.1.473 |
0.644 |
|
2007 |
Moldt B, Yant SR, Andersen PR, Kay MA, Mikkelsen JG. Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors. Human Gene Therapy. 18: 1193-204. PMID 17988194 DOI: 10.1089/Hum.2007.099 |
0.493 |
|
2007 |
Garrison BS, Yant SR, Mikkelsen JG, Kay MA. Postintegrative gene silencing within the Sleeping Beauty transposition system. Molecular and Cellular Biology. 27: 8824-33. PMID 17938204 DOI: 10.1128/Mcb.00498-07 |
0.799 |
|
2007 |
Kay MA. AAV vectors and tumorigenicity. Nature Biotechnology. 25: 1111-3. PMID 17921994 DOI: 10.1038/Nbt1007-1111 |
0.335 |
|
2007 |
Paskowitz DM, Greenberg KP, Yasumura D, Grimm D, Yang H, Duncan JL, Kay MA, Lavail MM, Flannery JG, Vollrath D. Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium. Human Gene Therapy. 18: 871-80. PMID 17892416 DOI: 10.1089/Hum.2007.065 |
0.648 |
|
2007 |
Tward AD, Jones KD, Yant S, Cheung ST, Fan ST, Chen X, Kay MA, Wang R, Bishop JM. Distinct pathways of genomic progression to benign and malignant tumors of the liver. Proceedings of the National Academy of Sciences of the United States of America. 104: 14771-6. PMID 17785413 DOI: 10.1073/Pnas.0706578104 |
0.335 |
|
2007 |
Shen X, Storm T, Kay MA. Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1955-62. PMID 17726459 DOI: 10.1038/Sj.Mt.6300293 |
0.473 |
|
2007 |
Inagaki K, Ma C, Storm TA, Kay MA, Nakai H. The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. Journal of Virology. 81: 11304-21. PMID 17686847 DOI: 10.1128/Jvi.01225-07 |
0.345 |
|
2007 |
Inagaki K, Lewis SM, Wu X, Ma C, Munroe DJ, Fuess S, Storm TA, Kay MA, Nakai H. DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice. Journal of Virology. 81: 11290-303. PMID 17686840 DOI: 10.1128/Jvi.00963-07 |
0.456 |
|
2007 |
Azuma H, Paulk N, Ranade A, Dorrell C, Al-Dhalimy M, Ellis E, Strom S, Kay MA, Finegold M, Grompe M. Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice. Nature Biotechnology. 25: 903-10. PMID 17664939 DOI: 10.1038/Nbt1326 |
0.387 |
|
2007 |
Riu E, Chen ZY, Xu H, He CY, Kay MA. Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1348-55. PMID 17457320 DOI: 10.1038/Sj.Mt.6300177 |
0.405 |
|
2007 |
Zayed H, Xia L, Yerich A, Yant SR, Kay MA, Puttaraju M, McGarrity GJ, Wiest DL, McIvor RS, Tolar J, Blazar BR. Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1273-9. PMID 17457319 DOI: 10.1038/Sj.Mt.6300178 |
0.407 |
|
2007 |
Yant SR, Huang Y, Akache B, Kay MA. Site-directed transposon integration in human cells. Nucleic Acids Research. 35: e50. PMID 17344320 DOI: 10.1093/Nar/Gkm089 |
0.403 |
|
2007 |
Grimm D, Kay MA. Combinatorial RNAi: a winning strategy for the race against evolving targets? Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 878-88. PMID 17311009 DOI: 10.1038/Sj.Mt.6300116 |
0.626 |
|
2007 |
Akache B, Grimm D, Shen X, Fuess S, Yant SR, Glazer DS, Park J, Kay MA. A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 330-9. PMID 17235311 DOI: 10.1038/Sj.Mt.6300053 |
0.702 |
|
2007 |
Wang AY, Ehrhardt A, Xu H, Kay MA. Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 255-63. PMID 17235302 DOI: 10.1038/Sj.Mt.6300032 |
0.615 |
|
2007 |
Ehrhardt A, Yant SR, Giering JC, Xu H, Engler JA, Kay MA. Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 146-56. PMID 17164786 DOI: 10.1038/Sj.Mt.6300011 |
0.819 |
|
2007 |
Tolar J, Nauta AJ, Osborn MJ, Panoskaltsis Mortari A, McElmurry RT, Bell S, Xia L, Zhou N, Riddle M, Schroeder TM, Westendorf JJ, McIvor RS, Hogendoorn PC, Szuhai K, Oseth L, ... ... Kay MA, et al. Sarcoma derived from cultured mesenchymal stem cells. Stem Cells (Dayton, Ohio). 25: 371-9. PMID 17038675 DOI: 10.1634/Stemcells.2005-0620 |
0.343 |
|
2007 |
Zhang X, Epperly MW, Kay MA, Chen Z, Smith T, Franicola D, Greenberger B, Komanduri P, Greenberger JS. Minicircle Plasmid Containing the Human Manganese Superoxide Dismutase (MnSOD) Transgene Confers Radioprotection to Hematopoietic Progenitor Cell Line 32Dcl3. Blood. 110: 5138-5138. DOI: 10.1182/Blood.V110.11.5138.5138 |
0.409 |
|
2007 |
Kay MA. MicroRNAs outwit immune limitations in gene therapy Blood. 110: 4136-4137. DOI: 10.1182/Blood-2007-09-111062 |
0.453 |
|
2007 |
Zhang X, Epperly M, Kay M, Chen Z, Smith T, Franicola D, Greenberger J. A Minicircle Plasmid Containing the Human Manganese Superoxide Dismutase (MnSOD) Transgene Confers Radioprotection to Cells InVitro International Journal of Radiation Oncology*Biology*Physics. 69: S625. DOI: 10.1016/J.Ijrobp.2007.07.1952 |
0.364 |
|
2006 |
Ehrhardt A, Engler JA, Xu H, Cherry AM, Kay MA. Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration. Human Gene Therapy. 17: 1077-94. PMID 17069535 DOI: 10.1089/Hum.2006.17.1077 |
0.34 |
|
2006 |
Akache B, Grimm D, Pandey K, Yant SR, Xu H, Kay MA. The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. Journal of Virology. 80: 9831-6. PMID 16973587 DOI: 10.1128/Jvi.00878-06 |
0.622 |
|
2006 |
Grimm D, Streetz KL, Jopling CL, Storm TA, Pandey K, Davis CR, Marion P, Salazar F, Kay MA. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature. 441: 537-41. PMID 16724069 DOI: 10.1038/Nature04791 |
0.645 |
|
2006 |
Inagaki K, Fuess S, Storm TA, Gibson GA, Mctiernan CF, Kay MA, Nakai H. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Molecular Therapy : the Journal of the American Society of Gene Therapy. 14: 45-53. PMID 16713360 DOI: 10.1016/J.Ymthe.2006.03.014 |
0.467 |
|
2006 |
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Rasko J, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, ... ... Kay MA, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature Medicine. 12: 342-7. PMID 16474400 DOI: 10.1038/Nm1358 |
0.401 |
|
2006 |
Grimm D, Kay MA. Therapeutic short hairpin RNA expression in the liver: viral targets and vectors. Gene Therapy. 13: 563-75. PMID 16453009 DOI: 10.1038/Sj.Gt.3302727 |
0.576 |
|
2006 |
Tolar J, O'shaughnessy MJ, Panoskaltsis-Mortari A, McElmurry RT, Bell S, Riddle M, McIvor RS, Yant SR, Kay MA, Krause D, Verfaillie CM, Blazar BR. Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells. Blood. 107: 4182-8. PMID 16410448 DOI: 10.1182/Blood-2005-08-3289 |
0.351 |
|
2006 |
Grimm D, Pandey K, Nakai H, Storm TA, Kay MA. Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. Journal of Virology. 80: 426-39. PMID 16352567 DOI: 10.1128/Jvi.80.1.426-439.2006 |
0.659 |
|
2006 |
Tolar J, Nauta AJ, Osborn MJ, Panoskaltsis-Mortari A, McElmurry RT, Bell S, Xia L, Zhou N, Riddle M, Schroeder TM, Westendorf JJ, McIvor RS, Hogendoom PC, Szuhai K, Oseth L, ... ... Kay MA, et al. Osteosarcoma Derived from Cultured Mesenchymal Stem Cells. Blood. 108: 2554-2554. DOI: 10.1182/Blood.V108.11.2554.2554 |
0.342 |
|
2006 |
Cao D, Kay MA. 852. Chromatin Study of Adenovirus Vector DNA In Vivo Molecular Therapy. 13: S329. DOI: 10.1016/J.Ymthe.2006.08.938 |
0.414 |
|
2006 |
Giering JC, Grimm D, Storm TA, Xu H, Kay MA. 796. Pol II-Driven shRNA as an Effective Hepatitis B Virus Therapeutic Molecular Therapy. 13: S308-S309. DOI: 10.1016/J.Ymthe.2006.08.885 |
0.799 |
|
2006 |
Garrison BS, Yant SR, Mikkelsen JG, Kay MA. 792. Post-Integrative Gene Silencing in the Sleeping Beauty Transposition System Molecular Therapy. 13: S307. DOI: 10.1016/J.Ymthe.2006.08.880 |
0.796 |
|
2006 |
Shen X, Xu H, Storm T, Kay MA. 746. Localization of Structural Determinants in AAV Capsid for Efficient Liver Transduction by Domain Swapping between AAV-2 and AAV-8 Molecular Therapy. 13: S288. DOI: 10.1016/J.Ymthe.2006.08.828 |
0.417 |
|
2006 |
Grimm D, Lee JS, Storm TA, Kay MA. 742. Molecular Evolution of Adeno-Associated Viral (AAV) Vectors Via DNA Family Shuffling of Primate and Non-Primate Serotypes Molecular Therapy. 13: S287. DOI: 10.1016/J.Ymthe.2006.08.824 |
0.656 |
|
2006 |
Xie L, Kang Y, Yant S, Kay M, McCray P. 680. Non-Viral Transposon Mediated Gene Transfer of Human Factor VIII to Hemophilia A Mice Molecular Therapy. 13: S262. DOI: 10.1016/J.Ymthe.2006.08.757 |
0.443 |
|
2006 |
Riu E, Chen Z, Kay MA. 539. Unraveling the Mechanisms Underlying Silencing/Activation of Episomal Vectors In Vivo Molecular Therapy. 13: S207. DOI: 10.1016/J.Ymthe.2006.08.610 |
0.45 |
|
2006 |
Chen Z, He C, Ehrhardt A, Xu H, Kay MA. 516. Development of a Minicircle Vector Free of Plasmid Bacterial DNA Sequences and Capable of øC31-Mediated Site-Specific Integration Molecular Therapy. 13: S198-S199. DOI: 10.1016/J.Ymthe.2006.08.587 |
0.472 |
|
2006 |
Hebert ML, Grimm D, Storm TA, Kay MA. 489. Treatment for Hemophilia B Using Self- Complimentary AAV8 Vectors Molecular Therapy. 13: S189-S190. DOI: 10.1016/J.Ymthe.2006.08.559 |
0.677 |
|
2006 |
Ehrhardt A, Engler JA, Xu H, Cherry A, Kay MA. 388. Chromosomal Rearrangements in Mammalian Cells Are Induced at High Frequency by the Bacteriophage Derived Integrase phiC31 Molecular Therapy. 13: S148. DOI: 10.1016/J.Ymthe.2006.08.449 |
0.347 |
|
2006 |
Haussecker D, Elazar M, Glenn JS, Kay MA. 374. Hepatitis Delta Virus-Mediated Amplification of Therapeutic RNAi Molecular Therapy. 13: S142. DOI: 10.1016/J.Ymthe.2006.08.433 |
0.453 |
|
2006 |
Inagaki K, Storm TA, Kay MA, Nakai H. 114. Revisiting rAAV Vector Integration in scid Mice: DNA-PKcs Deficiency Does Not Substantially Increase Integration Frequency in Hepatic and Non-Hepatic Tissues In Vivo Molecular Therapy. 13: S47. DOI: 10.1016/J.Ymthe.2006.08.134 |
0.437 |
|
2006 |
Shen X, Xu H, Storm T, Kay MA. 108. Study of an AAV-8 Capsid Mutant with Direct Heparin Binding Capability but Reduced Efficiency in Liver-Targeted Transduction Molecular Therapy. 13: S44-S45. DOI: 10.1016/J.Ymthe.2006.08.128 |
0.449 |
|
2006 |
Akache B, Fuess S, Grimm D, Kay MA. 103. A Screen for Host Cellular Proteins That Interact with Adeno-Associated Virus Capsid Proteins Reveals Proteins Involved in AAV8 Transduction Molecular Therapy. 13: S42-S43. DOI: 10.1016/J.Ymthe.2006.08.123 |
0.607 |
|
2006 |
Grimm D, Streetz KL, Storm TA, Jopling CL, Lee JS, Kay MA. 805. Fatality in Mice Due to Oversaturation of Cellular Micro/Short Hairpin RNA Pathways Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.1226 |
0.672 |
|
2006 |
Wursthorn K, Storm T, Kay MA, Finegold M, Grompe M. 804. In Vivo Correction of a Metabolic Liver Disease by AAV8-Mediated Homologous Recombination Molecular Therapy. 13: S311-S312. DOI: 10.1016/J.Ymthe.2006.08.1225 |
0.458 |
|
2006 |
Couto LB, Parker AE, Haniff G, Suhy DA, Kolykhalov AA, Roelvink PW, Maria Garcia L, Schroeder R, Kay MA, Cunningham SM. 1099. RNAi-Based Therapy for the Treatment of HCV Molecular Therapy. 13: S422-S423. DOI: 10.1016/J.Ymthe.2006.08.1203 |
0.365 |
|
2006 |
Grimm D, Lee JS, Streetz KL, Storm TA, Kay MA. 1066. A Novel Class of Miniature Stabilized Double-Stranded AAV (msdsAAV) Vectors for the In Vivo Expression of Short Hairpin RNAs Molecular Therapy. 13: S409. DOI: 10.1016/J.Ymthe.2006.08.1164 |
0.636 |
|
2006 |
Suhy DA, Kolykhalov AA, Couto LB, Garcia L, Schroeder AR, Parker AE, Haniff G, Kay MA, Roelvink P. 1034. Expression of Short Hairpin RNAs by Liver and Non Liver Specific RNA Pol II Expression Cassettes: What Governs Activity? Molecular Therapy. 13: S397. DOI: 10.1016/J.Ymthe.2006.08.1129 |
0.443 |
|
2006 |
Ehrhardt A, Xu H, Dillow AM, Yant SR, Nichols TC, Kay MA. 11. Transposition from a Gene-Deleted Adenoviral Vector Results in Phenotypic Correction in a Canine Model for Hemophilia B Molecular Therapy. 13: S5. DOI: 10.1016/J.Ymthe.2006.08.022 |
0.49 |
|
2006 |
Inagaki K, Fuess S, Storm TA, Kay MA, Nakai H. 9. Biology of rAAV8 in Mouse Liver Following Vector Administration at Birth Molecular Therapy. 13: S4. DOI: 10.1016/J.Ymthe.2006.08.019 |
0.442 |
|
2006 |
Inagaki K, Storm TA, Kay MA, Nakai H. 5. Mechanisms for Hairpin Loop Opening of “Closed” AAV-ITRs by Specific Cellular Endonuclease Activities, a Prerequisite for rAAV Vector Genome Recombinations In Vivo Molecular Therapy. 13: S2-S3. DOI: 10.1016/J.Ymthe.2006.08.015 |
0.415 |
|
2005 |
Ehrhardt A, Kay MA. Gutted adenovirus: a rising star on the horizon? Gene Therapy. 12: 1540-1. PMID 18360947 DOI: 10.1038/Sj.Gt.3302597 |
0.51 |
|
2005 |
Tward AD, Jones KD, Yant S, Kay MA, Wang R, Bishop JM. Genomic progression in mouse models for liver tumors. Cold Spring Harbor Symposia On Quantitative Biology. 70: 217-24. PMID 16869757 DOI: 10.1101/Sqb.2005.70.058 |
0.342 |
|
2005 |
Ohashi K, Kay MA, Yokoyama T, Kuge H, Kanehiro H, Hisanaga M, Ko S, Nakajima Y. Stability and repeat regeneration potential of the engineered liver tissues under the kidney capsule in mice. Cell Transplantation. 14: 621-7. PMID 16405072 DOI: 10.3727/000000005783982620 |
0.348 |
|
2005 |
Tolar J, Osborn M, Bell S, McElmurry R, Xia L, Riddle M, Panoskaltsis-Mortari A, Jiang Y, McIvor RS, Contag CH, Yant SR, Kay MA, Verfaillie CM, Blazar BR. Real-time in vivo imaging of stem cells following transgenesis by transposition. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 42-8. PMID 15963919 DOI: 10.1016/J.Ymthe.2005.02.023 |
0.377 |
|
2005 |
Riu E, Grimm D, Huang Z, Kay MA. Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo. Human Gene Therapy. 16: 558-70. PMID 15916481 DOI: 10.1089/Hum.2005.16.558 |
0.661 |
|
2005 |
Ehrhardt A, Xu H, Huang Z, Engler JA, Kay MA. A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase. Molecular Therapy : the Journal of the American Society of Gene Therapy. 11: 695-706. PMID 15851008 DOI: 10.1016/J.Ymthe.2005.01.010 |
0.509 |
|
2005 |
Ohashi K, Nakai H, Couto LB, Kay MA. Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver. Human Gene Therapy. 16: 299-306. PMID 15812225 DOI: 10.1089/Hum.2005.16.299 |
0.469 |
|
2005 |
Yant SR, Wu X, Huang Y, Garrison B, Burgess SM, Kay MA. High-resolution genome-wide mapping of transposon integration in mammals. Molecular and Cellular Biology. 25: 2085-94. PMID 15743807 DOI: 10.1128/Mcb.25.6.2085-2094.2005 |
0.779 |
|
2005 |
Nakai H, Wu X, Fuess S, Storm TA, Munroe D, Montini E, Burgess SM, Grompe M, Kay MA. Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. Journal of Virology. 79: 3606-14. PMID 15731255 DOI: 10.1128/Jvi.79.6.3606-3614.2005 |
0.44 |
|
2005 |
Chen ZY, He CY, Kay MA. Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo. Human Gene Therapy. 16: 126-31. PMID 15703495 DOI: 10.1089/Hum.2005.16.126 |
0.46 |
|
2005 |
Grimm D, Pandey K, Kay MA. Adeno-associated virus vectors for short hairpin RNA expression. Methods in Enzymology. 392: 381-405. PMID 15644194 DOI: 10.1016/S0076-6879(04)92023-X |
0.659 |
|
2005 |
Ohashi K, Waugh JM, Dake MD, Yokoyama T, Kuge H, Nakajima Y, Yamanouchi M, Naka H, Yoshioka A, Kay MA. Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases. Hepatology (Baltimore, Md.). 41: 132-40. PMID 15619229 DOI: 10.1002/Hep.20484 |
0.361 |
|
2005 |
Nakai H, Fuess S, Storm TA, Muramatsu S, Nara Y, Kay MA. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. Journal of Virology. 79: 214-24. PMID 15596817 DOI: 10.1128/Jvi.79.1.214-224.2005 |
0.461 |
|
2005 |
Tolar J, Osborn MJ, Panoskaltsis-Mortari A, McElmurry RT, Bell S, Xia L, Riddle M, Schroeder TM, Westendorf JJ, McIvor RS, Oseth L, Hirsch B, Yant SR, Kay MA, Peister A, et al. Mesenchymal Cancer Cells Can Arise from Ex Vivo Modified Mesenchymal Stem Cells. Blood. 106: 4326-4326. DOI: 10.1182/Blood.V106.11.4326.4326 |
0.346 |
|
2005 |
Yant SR, Huang Y, Kay MA. 1101. Fusion Proteins Consisting of the Sleeping Beauty Transposase and the Polydactyl Zinc Finger Protein E2C Direct Transposon Integration into a Unique Human Chromosomal Sequence Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.649 |
0.395 |
|
2005 |
Akache B, Glazer D, Park J, Kay MA. 1084. A Screen for Host Cellular Proteins That Interact with Adeno-Associated Virus Capsid Proteins Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.631 |
0.573 |
|
2005 |
Noll M, Bennett R, Yant S, Kay MA, Grompe M. 1046. Stable Correction of Hematopoietic Stem Cells with Non-Viral Gene Transfer Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.593 |
0.439 |
|
2005 |
Suhy DA, Couto LB, Kolykhalov AA, Parker AE, Schroeder AR, Garcia LM, Haniff G, Cunningham SM, Kay MA, Roelvink PW. 1000. In Vitro and In Vivo Evaluation of Optimized Short-Hairpin RNAs for the Treatment of the Hepatitis C Virus Using Expression Cassettes Producing One, Two or Three Anti HCV shRNAs Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.547 |
0.326 |
|
2005 |
Kolykhalov AA, Graham MW, Suhy DA, Roelvink PW, Schroeder AR, Parker AE, Garcia LM, Kay MA, Cunningham SM, Couto LB. 997. Loop Composition as Well as Orientation of the Guide Strand in shRNA May Dramatically Modulate Activity of the shRNA: Effect on the Development of RNAi-Based Inhibitors Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.544 |
0.344 |
|
2005 |
Grimm D, Pandey K, Streetz KL, Kay MA. 857. A Versatile and Efficient Viral Vector Platform for Stable In Vivo Expression of Short Hairpin RNA Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.400 |
0.684 |
|
2005 |
Ehrhardt A, Yant SR, Jeff GA, Xu H, Calos MP, Kay MA. 821. Development of a Site-Specific Integrating Gene-Deleted Adenoviral Vector for Sustained Transgene Expression Levels In Vivo Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.364 |
0.472 |
|
2005 |
Ehrhardt A, Xu H, Yant SR, Engler JA, Kay MA. 600. A Novel Gene-Deleted Adenoviral Vector for Somatic Integration and Long-Term Phenotypic Correction of Murine and Canine Hemophilia B Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.140 |
0.463 |
|
2005 |
Ohashi K, Kay MA, Yokoyama T, Kuge H, Kanehiro H, Hisanaga M, Ko S, Naka H, Yoshioka A, Nakajima Y. 597. Liver Tissue Engineering at Extra-Hepatic Site: A Novel Therapeutic Approach toward Hemophilia Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.137 |
0.351 |
|
2005 |
Garrison BS, Yant SR, Mikkelsen JG, Kay MA. 573. Investigation into the Frequency of Post-Integrative Gene Silencing Using a Non-Selective Sleeping Beauty Transposition System Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.113 |
0.796 |
|
2005 |
Riu E, Chen Z, Kay MA. 563. Differential Histone Modifications in Transcriptional Active Versus Silenced Episomal Vectors In Vivo Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.103 |
0.455 |
|
2005 |
Chen Z, He C, Xu H, Kay MA. 557. Unraveling the Molecular Mechanisms Responsible for Episomal Transgene Silencing In Vivo Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.097 |
0.376 |
|
2005 |
Inagaki K, Wu X, Fuess S, Storm TA, Kay MA, Nakai H. 516. Analysis of AAV Serotype 8 Vector Integration in Normal and DNA-PKcs-Deficient Scid Mice by a Novel Strategy Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.056 |
0.456 |
|
2005 |
Inagaki K, Storm TA, Fuess S, Kay MA, Nakai H. 504. In Vivo Characterization of AAV Serotype 9 Vectors in Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.044 |
0.444 |
|
2005 |
Suhy DA, Kolykhalov AA, Couto LB, Garcia LM, Schroeder AR, Parker AE, Haniff G, Cunningham SM, Kay MA, Roelvink PW. 443. Tissue Specific Expression of Short Hairpin RNAs for the Treatment of HCV Using Liver Specific RNA Pol II Expression Cassettes Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.451 |
0.417 |
|
2005 |
Ehrhardt A, Xu H, Salazar FH, Marion PL, Kay MA. 441. Short Hairpin RNA Interference Expressed from a Gene-Deleted Adenoviral Vector Results in Reduction of Hepatits B Surface Antigen Levels In Vitro and in a Small Animal Model for Hepatitis B Infection Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.449 |
0.512 |
|
2005 |
Roelvink PW, Graham MW, Suhy DA, Couto LB, Cunningham SM, Kay MA, Rossi JJ, Kolykhalov AA. 437. Alternative Strategies To Deliver Multiple shRNAs in Order to Simultaneously Inhibit Multiple Targets Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.445 |
0.438 |
|
2005 |
Shen X, Xu H, Huang Z, Storm TA, Kay MA. 405. Comparison Study of Structural Determinants for AAV Liver Transduction by Domain Swapping between AAV-2 and AAV-8 Capsids Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.408 |
0.499 |
|
2005 |
Park J, Yant SR, Kay MA. 191. Identification of a Novel Functional Domain in the Sleeping Beauty Transposase: Towards Alleviating the Restriction of SB Overproduction Inhibition Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.194 |
0.531 |
|
2005 |
Giering JC, Kay MA. 88. Driving shRNA Transcription from PolII Promoters in Mammalian Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.066 |
0.807 |
|
2005 |
Grimm D, Streetz KL, Pandey K, Storm TA, Kay MA. 87. Determinants of Toxicity Induced by In Vivo Expression of Short Hairpin RNA from AAV Vectors Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.065 |
0.687 |
|
2005 |
Mikkelsen JG, Yant SR, Ehrhardt A, Kay MA. 22. Generation of Circular Sleeping Beauty DNA Transposition Substrates by RU486-Induced Flp Recombination In Vitro |[ndash]| Steps towards a Single Integrating Adeno-Transposon Vector Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.025 |
0.536 |
|
2004 |
Yant SR, Park J, Huang Y, Mikkelsen JG, Kay MA. Mutational analysis of the N-terminal DNA-binding domain of sleeping beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells. Molecular and Cellular Biology. 24: 9239-47. PMID 15456893 DOI: 10.1128/Mcb.24.20.9239-9247.2004 |
0.529 |
|
2004 |
Jenkins DD, Streetz K, Tataria M, Sahar D, Kurobe M, Longaker MT, Kay MA, Sylvester KG. Donor-derived, liver-specific protein expression after bone marrow transplantation. Transplantation. 78: 530-6. PMID 15446311 DOI: 10.1097/01.Tp.0000130180.42573.B1 |
0.393 |
|
2004 |
Layzer JM, McCaffrey AP, Tanner AK, Huang Z, Kay MA, Sullenger BA. In vivo activity of nuclease-resistant siRNAs. Rna (New York, N.Y.). 10: 766-71. PMID 15100431 DOI: 10.1261/Rna.5239604 |
0.425 |
|
2004 |
Wang AY, Peng PD, Ehrhardt A, Storm TA, Kay MA. Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo. Human Gene Therapy. 15: 405-13. PMID 15053865 DOI: 10.1089/104303404322959551 |
0.681 |
|
2004 |
Chen ZY, He CY, Meuse L, Kay MA. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Therapy. 11: 856-64. PMID 15029228 DOI: 10.1038/Sj.Gt.3302231 |
0.428 |
|
2004 |
Thomas CE, Storm TA, Huang Z, Kay MA. Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. Journal of Virology. 78: 3110-22. PMID 14990730 DOI: 10.1128/Jvi.78.6.3110-3122.2004 |
0.439 |
|
2004 |
Tolar J, Bell S, McElmurry R, Xia L, McIvor RS, Yant SR, Kay MA, Contag CH, Verfaillie CM, Blazar BR. Real-Time In Vivo Biodistribution of Multipotent Adult Progenitor Cells (MAPC): Role of the Immune System in MAPC Resistance in Non-Transplanted and Bone Marrow Transplanted Mice. Blood. 104: 507-507. DOI: 10.1182/Blood.V104.11.507.507 |
0.325 |
|
2004 |
High K, Tigges M, Manno C, Sabatino D, Arruda V, Herzog R, Rustagi P, Rasko J, Sommer J, Jaworski K, Ragni M, Glader B, Lessard R, Luk A, Couto L, ... ... Kay M, et al. Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B. Blood. 104: 413-413. DOI: 10.1182/Blood.V104.11.413.413 |
0.382 |
|
2004 |
Tolar J, Osborn M, Bell S, Xia L, Riddle M, Panoskaltsis-Mortari A, McIvor S, Stephen YR, Kay MA, Contag CH, Verfaillie CM, Blazar BR. Transgenesis of Multipotent Adult Progenitor Cells (MAPC) with Sleeping Beauty Transposons to Determine MAPC Homing and Persistence in Real-Time In Vivo. Blood. 104: 2099-2099. DOI: 10.1182/Blood.V104.11.2099.2099 |
0.498 |
|
2004 |
Riu E, Huang Z, Kay MA. 1039. Mechanistic Insights into the Persistence of Non-Viral Mediated Gene Transfer In Vivo Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.984 |
0.534 |
|
2004 |
High K, Manno C, Sabatino D, Hutchison S, Dake M, Razavi M, Kaye R, Aruda V, Herzog R, Rustagi P, Rasko J, Hoots K, Blatt P, Sommer J, Ragni M, ... ... Kay M, et al. 1002. Immune Responses to AAV and to Factor IX in a Phase I Study of AAV-Mediated, Liver-Directed Gene Transfer for Hemophilia B Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.940 |
0.392 |
|
2004 |
Grimm D, Streetz KL, Storm TA, Nakai H, McCaffrey AP, Huang Z, Salazar FH, Marion PL, Kay MA. 371. Complete Inhibition of Hepatitis B Virus Gene Expression In Vivo with Short Hairpin RNA Expressed from a Novel Double-Stranded, Bi-Cistronic Adeno-Associated Virus Pseudotype 8 Vector Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.918 |
0.659 |
|
2004 |
Ohashi K, Yokoyama T, Kuge H, Nakajima Y, Kay MA. 876. Towards establishing a heterologous liver in rodents Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.847 |
0.355 |
|
2004 |
Noll M, Bennett R, Yant S, Mikkelsen JG, Chen C, Kay MA, Grompe M. 826. Non-Viral Gene Therapy Targeting Fanconi Anemia Hematopoietic Stem Cells: A Realistic Hope? Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.723 |
0.453 |
|
2004 |
Yant SR, Park J, Huang Y, Mikkelsen JG, Kay MA. 819. Hyperactive Transposase Mutants of the Sleeping Beauty Transposon Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.717 |
0.616 |
|
2004 |
Yant SR, Wu X, Huang Y, Daigle B, Garrison BA, Burgess SM, Kay MA. 817. Nonrandom Insertion Site Preferences for the SB Transposon In Vitro and In Vivo Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.715 |
0.44 |
|
2004 |
Garrison BS, Yant SR, Mikkelsen JG, Kay MA. 815. Investigation into the Frequency of Post-Integrative Gene Silencing Using a Non-Selective Sleeping Beauty Transposition System Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.713 |
0.787 |
|
2004 |
Ehrhardt A, Engler JA, Calos MP, Kay MA. 811. The Hotspots of Integration Targeted by the Site-Specific Integrase PhiC31 May Be Cell Line Dependent Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.709 |
0.41 |
|
2004 |
Nakai H, Storm T, Fuess S, Kay MA. 337. Unrestricted Liver Transduction with AAV Serotype 8 Vector in Mice Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.279 |
0.447 |
|
2004 |
Streetz KL, Jenkins DD, Longaker MT, Sylvester KG, Kay MA. 308. A New Model to Functionally Measure Fusion Events in the Liver After Bone-Marrow Transplantation Over Time Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.250 |
0.314 |
|
2004 |
Wang AY, Peng PD, Ehrhardt A, Storm TA, Kay MA. 172. Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.132 |
0.68 |
|
2004 |
Park J, Yant SR, Kay MA. 148. The altered binding properties of sleeping beauty transposase hyperactive mutants may explain their enhanced efficacy Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.072 |
0.522 |
|
2004 |
Chen Z, He C, Kay MA. 141. Development of a one-step column purification of minicircle vector DNA devoid of bacterial sequences that results in high level persistent transgene expression in vivo Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.057 |
0.473 |
|
2004 |
Roelvink PW, Suhy DA, Kolykhalov AA, Cunningham SM, Mizutani T, Kay MA. 105. Optimization of Short-Hairpin RNAs for the Treatment of the Hepatitis C Virus Using AAV Vectors with Multiple Expression Cassettes Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.042 |
0.378 |
|
2004 |
Grimm D, Nakai H, Storm TA, Riu EP, Kay MA. 96. Transgene Expression from Recombinant AAV-8 Vectors in the Liver Occurs Independently of the Serotype Origin of the Viral Inverted Terminal Repeats Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.031 |
0.645 |
|
2004 |
Nakai H, Wu X, Fuess S, Storm T, Burgess S, Grompe M, Kay MA. 13. Hot Spots for rAAV2 Vector Integration in Mice Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.094 |
0.483 |
|
2004 |
McCaffrey AP, Pandey K, Ehrhardt A, Huang Z, Kay MA. 58. Toward Adenoviral Mediated RNA Interference for the Treatment of Hepatitis B Virus Infection|[ast]| Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.039 |
0.485 |
|
2003 |
Yant SR, Kay MA. Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells. Molecular and Cellular Biology. 23: 8505-18. PMID 14612396 DOI: 10.1128/Mcb.23.23.8505-8518.2003 |
0.306 |
|
2003 |
Mikkelsen JG, Yant SR, Meuse L, Huang Z, Xu H, Kay MA. Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Molecular Therapy : the Journal of the American Society of Gene Therapy. 8: 654-65. PMID 14529839 DOI: 10.1016/S1525-0016(03)00216-8 |
0.541 |
|
2003 |
McCaffrey A, Kay MA, Contag CH. Advancing molecular therapies through in vivo bioluminescent imaging. Molecular Imaging. 2: 75-86. PMID 12964305 DOI: 10.1162/153535003322331966 |
0.363 |
|
2003 |
Chyung YH, Peng PD, Kay MA. System for simultaneous tissue-specific and disease-specific regulation of therapeutic gene expression. Human Gene Therapy. 14: 1255-64. PMID 12952597 DOI: 10.1089/104303403767740795 |
0.47 |
|
2003 |
Chen ZY, He CY, Ehrhardt A, Kay MA. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Molecular Therapy : the Journal of the American Society of Gene Therapy. 8: 495-500. PMID 12946323 DOI: 10.1016/S1525-0016(03)00168-0 |
0.441 |
|
2003 |
Park F, Ohashi K, Kay MA. The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo. Molecular Therapy : the Journal of the American Society of Gene Therapy. 8: 314-23. PMID 12907154 DOI: 10.1016/S1525-0016(03)00169-2 |
0.442 |
|
2003 |
Bordier BB, Ohkanda J, Liu P, Lee SY, Salazar FH, Marion PL, Ohashi K, Meuse L, Kay MA, Casey JL, Sebti SM, Hamilton AD, Glenn JS. In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus. The Journal of Clinical Investigation. 112: 407-14. PMID 12897208 DOI: 10.1172/Jci17704 |
0.414 |
|
2003 |
McCaffrey AP, Meuse L, Karimi M, Contag CH, Kay MA. A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice. Hepatology (Baltimore, Md.). 38: 503-8. PMID 12883495 DOI: 10.1053/Jhep.2003.50330 |
0.342 |
|
2003 |
Grimm D, Kay MA. From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Current Gene Therapy. 3: 281-304. PMID 12871018 DOI: 10.2174/1566523034578285 |
0.647 |
|
2003 |
Nakai H, Storm TA, Fuess S, Kay MA. Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors. Human Gene Therapy. 14: 871-81. PMID 12828858 DOI: 10.1089/104303403765701169 |
0.374 |
|
2003 |
Ortiz-Urda S, Lin Q, Yant SR, Keene D, Kay MA, Khavari PA. Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer. Gene Therapy. 10: 1099-104. PMID 12808440 DOI: 10.1038/Sj.Gt.3301978 |
0.446 |
|
2003 |
Ehrhardt A, Xu H, Kay MA. Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo. Journal of Virology. 77: 7689-95. PMID 12805471 DOI: 10.1128/Jvi.77.13.7689-7695.2003 |
0.485 |
|
2003 |
Ehrhardt A, Xu H, Dillow AM, Bellinger DA, Nichols TC, Kay MA. A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood. 102: 2403-11. PMID 12805062 DOI: 10.1182/Blood-2003-01-0314 |
0.477 |
|
2003 |
Grimm D, Zhou S, Nakai H, Thomas CE, Storm TA, Fuess S, Matsushita T, Allen J, Surosky R, Lochrie M, Meuse L, McClelland A, Colosi P, Kay MA. Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood. 102: 2412-9. PMID 12791653 DOI: 10.1182/Blood-2003-02-0495 |
0.655 |
|
2003 |
Grimm D, Kay MA, Kleinschmidt JA. Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Molecular Therapy : the Journal of the American Society of Gene Therapy. 7: 839-50. PMID 12788658 DOI: 10.1016/S1525-0016(03)00095-9 |
0.746 |
|
2003 |
Nakai H, Montini E, Fuess S, Storm TA, Grompe M, Kay MA. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nature Genetics. 34: 297-302. PMID 12778174 DOI: 10.1038/Ng1179 |
0.5 |
|
2003 |
McCaffrey AP, Nakai H, Pandey K, Huang Z, Salazar FH, Xu H, Wieland SF, Marion PL, Kay MA. Inhibition of hepatitis B virus in mice by RNA interference. Nature Biotechnology. 21: 639-44. PMID 12740585 DOI: 10.1038/Nbt824 |
0.444 |
|
2003 |
Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nature Reviews. Genetics. 4: 346-58. PMID 12728277 DOI: 10.1038/Nrg1066 |
0.371 |
|
2003 |
Ehrhardt A, Peng PD, Xu H, Meuse L, Kay MA. Optimization of cis-acting elements for gene expression from nonviral vectors in vivo. Human Gene Therapy. 14: 215-25. PMID 12639302 DOI: 10.1089/10430340360535779 |
0.467 |
|
2003 |
Nakai H, Fuess S, Storm TA, Meuse LA, Kay MA. Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo Molecular Therapy. 7: 112-121. PMID 12573624 DOI: 10.1016/S1525-0016(02)00034-5 |
0.374 |
|
2003 |
Nakai H, Montini E, Fuess S, Storm TA, Meuse L, Finegold M, Grompe M, Kay MA. Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice Molecular Therapy. 7: 101-111. PMID 12573623 DOI: 10.1016/S1525-0016(02)00023-0 |
0.463 |
|
2003 |
Sclimenti CR, Neviaser AS, Baba EJ, Meuse L, Kay MA, Calos MP. Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice Biotechnology Progress. 19: 144-151. PMID 12573017 DOI: 10.1021/Bp0200907 |
0.555 |
|
2003 |
Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, ... ... Kay MA, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood. 101: 2963-72. PMID 12515715 DOI: 10.1182/Blood-2002-10-3296 |
0.443 |
|
2003 |
Jenkins D, Streetz K, Kay M, Longaker M, Sylvester K. A model to quantitate cell transplant function after liver-specific injury Journal of Surgical Research. 114: 305-306. DOI: 10.1016/J.Jss.2003.08.115 |
0.364 |
|
2002 |
Montini E, Held PK, Noll M, Morcinek N, Al-Dhalimy M, Finegold M, Yant SR, Kay MA, Grompe M. In vivo correction of murine tyrosinemia type I by DNA-mediated transposition. Molecular Therapy : the Journal of the American Society of Gene Therapy. 6: 759-69. PMID 12498772 DOI: 10.1006/Mthe.2002.0812 |
0.503 |
|
2002 |
McCaffrey AP, Kay MA. A story of mice and men. Gene Therapy. 9: 1563. PMID 12424608 DOI: 10.1038/Sj.Gt.3301890 |
0.445 |
|
2002 |
Nakai H, Thomas CE, Storm TA, Fuess S, Powell S, Wright JF, Kay MA. A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. Journal of Virology. 76: 11343-9. PMID 12388694 DOI: 10.1128/Jvi.76.22.11343-11349.2002 |
0.421 |
|
2002 |
Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP. Site-specific genomic integration produces therapeutic factor IX levels in mice Nature Biotechnology. 20: 1124-1128. PMID 12379870 DOI: 10.1038/Nbt753 |
0.436 |
|
2002 |
Yant SR, Ehrhardt A, Mikkelsen JG, Meuse L, Pham T, Kay MA. Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nature Biotechnology. 20: 999-1005. PMID 12244327 DOI: 10.1038/Nbt738 |
0.541 |
|
2002 |
Bordier BB, Marion PL, Ohashi K, Kay MA, Greenberg HB, Casey JL, Glenn JS. A prenylation inhibitor prevents production of infectious hepatitis delta virus particles. Journal of Virology. 76: 10465-72. PMID 12239323 DOI: 10.1128/Jvi.76.20.10465-10472.2002 |
0.323 |
|
2002 |
Ohashi K, Park F, Schwall RH, Kay MA. Efficient gene transduction to cultured hepatocytes by HIV-1 derived lentiviral vector Transplantation Proceedings. 34: 1431-1433. PMID 12176427 DOI: 10.1016/S0041-1345(02)02916-0 |
0.498 |
|
2002 |
McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. RNA interference in adult mice. Nature. 418: 38-9. PMID 12097900 DOI: 10.1038/418038A |
0.376 |
|
2002 |
McCaffrey AP, Ohashi K, Meuse L, Shen S, Lancaster AM, Lukavsky PJ, Sarnow P, Kay MA. Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 5: 676-84. PMID 12027551 DOI: 10.1006/Mthe.2002.0600 |
0.367 |
|
2002 |
Ehrhardt A, Kay MA. A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood. 99: 3923-30. PMID 12010790 DOI: 10.1182/Blood.V99.11.3923 |
0.457 |
|
2002 |
Ohashi K, Park F, Kay MA. Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo Human Gene Therapy. 13: 653-663. PMID 11916488 DOI: 10.1089/10430340252837242 |
0.461 |
|
2002 |
Kay MA. Gene transfer for hemophilia and viral infection International Journal of Hematology. 76: 147-147. DOI: 10.1007/Bf03165228 |
0.474 |
|
2001 |
Arruda VR, Fields PA, Milner R, Wainwright L, De Miguel MP, Donovan PJ, Herzog RW, Nichols TC, Biegel JA, Razavi M, Dake M, Huff D, Flake AW, Couto L, Kay MA, et al. Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males. Molecular Therapy : the Journal of the American Society of Gene Therapy. 4: 586-92. PMID 11735343 DOI: 10.1006/Mthe.2001.0491 |
0.427 |
|
2001 |
Mizuguchi H, Kay MA, Hayakawa T. Approaches for generating recombinant adenovirus vectors Advanced Drug Delivery Reviews. 52: 165-176. PMID 11718941 DOI: 10.1016/S0169-409X(01)00215-0 |
0.379 |
|
2001 |
Ohashi K, Park F, Kay MA. Hepatocyte transplantation: Clinical and experimental application Journal of Molecular Medicine. 79: 617-630. PMID 11715065 DOI: 10.1007/S001090100260 |
0.328 |
|
2001 |
Vollrath D, Feng W, Duncan JL, Yasumura D, D'Cruz PM, Chappelow A, Matthes MT, Kay MA, LaVail MM. Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proceedings of the National Academy of Sciences of the United States of America. 98: 12584-9. PMID 11592982 DOI: 10.1073/Pnas.221364198 |
0.318 |
|
2001 |
Park F, Kay MA. Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo Molecular Therapy. 4: 164-173. PMID 11545606 DOI: 10.1006/Mthe.2001.0450 |
0.511 |
|
2001 |
Stoll SM, Sclimenti CR, Baba EJ, Meuse L, Kay MA, Calos MP. Epstein-Barr virus/human vector provides high-level, long-term expression of α1-antitrypsin in mice Molecular Therapy. 4: 122-129. PMID 11482983 DOI: 10.1006/Mthe.2001.0429 |
0.531 |
|
2001 |
Nakai H, Yant SR, Storm TA, Fuess S, Meuse L, Kay MA. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo Journal of Virology. 75: 6969-6976. PMID 11435577 DOI: 10.1128/Jvi.75.15.6969-6976.2001 |
0.484 |
|
2001 |
Mizuguchi H, Koizumi N, Hosono T, Utoguchi N, Watanabe Y, Kay MA, Hayakawa T. A simplified system for constructing recombinant adenoviral vectors containing heterologous peptides in the HI loop of their fiber knob Gene Therapy. 8: 730-735. PMID 11406768 DOI: 10.1038/Sj.Gt.3301453 |
0.361 |
|
2001 |
Mizuguchi H, Kay MA, Hayakawa T. In vitro ligation-based cloning of foreign DNAs into the E3 and E1 deletion regions for generation of recombinant adenovirus vectors Biotechniques. 30: 1112-1116. PMID 11355346 DOI: 10.2144/01305Dd03 |
0.323 |
|
2001 |
Chen ZY, Yant SR, He CY, Meuse L, Shen S, Kay MA. Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver Molecular Therapy. 3: 403-410. PMID 11273783 DOI: 10.1006/Mthe.2001.0278 |
0.474 |
|
2001 |
Ohashi K, Meuse L, Schwall R, Kay MA. cMet activation allows persistent engraftment of ectopically transplanted xenogenic human hepatocytes in mice Transplantation Proceedings. 33: 587-588. PMID 11266970 DOI: 10.1016/S0041-1345(00)02154-0 |
0.352 |
|
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 |
0.445 |
|
2000 |
Nakai H, Storm TA, Kay MA. Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo Journal of Virology. 74: 9451-9463. PMID 11000214 DOI: 10.1128/Jvi.74.20.9451-9463.2000 |
0.437 |
|
2000 |
Miao CH, Ohashi K, Patijn GA, Meuse L, Ye X, Thompson AR, Kay MA. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Molecular Therapy : the Journal of the American Society of Gene Therapy. 1: 522-32. PMID 10933977 DOI: 10.1006/Mthe.2000.0075 |
0.499 |
|
2000 |
Park F, Ohashi K, Kay MA. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver Blood. 96: 1173-1176. PMID 10910939 DOI: 10.1182/Blood.V96.3.1173 |
0.447 |
|
2000 |
Yant SR, Meuse L, Chiu W, Ivics Z, Izsvak Z, Kay MA. Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system Nature Genetics. 25: 35-41. PMID 10802653 DOI: 10.1038/75568 |
0.503 |
|
2000 |
Nakai H, Storm TA, Kay MA. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors Nature Biotechnology. 18: 527-532. PMID 10802620 DOI: 10.1038/75390 |
0.529 |
|
2000 |
Miao CH, Nakai H, Thompson AR, Storm TA, Chiu W, Snyder RO, Kay MA. Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction. Journal of Virology. 74: 3793-803. PMID 10729154 DOI: 10.1128/Jvi.74.8.3793-3803.2000 |
0.53 |
|
2000 |
Ohashi K, Marion PL, Nakai H, Meuse L, Cullen JM, Bordier BB, Schwall R, Greenberg HB, Glenn JS, Kay MA. Sustained survival of human hepatocytes in mice: A model for in vivo infection with human hepatitis B and hepatitis delta viruses. Nature Medicine. 6: 327-31. PMID 10700236 DOI: 10.1038/73187 |
0.405 |
|
2000 |
Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature Genetics. 24: 257-61. PMID 10700178 DOI: 10.1038/73464 |
0.426 |
|
2000 |
Lieber A, Kay MA, Li ZY. Nuclear import of moloney murine leukemia virus DNA mediated by adenovirus preterminal protein is not sufficient for efficient retroviral transduction in nondividing cells Journal of Virology. 74: 721-734. PMID 10623734 DOI: 10.1128/Jvi.74.2.721-734.2000 |
0.372 |
|
1999 |
Lieber A, Steinwaerder DS, Carlson CA, Kay MA. Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes. Journal of Virology. 73: 9314-24. PMID 10516040 DOI: 10.1128/Jvi.73.11.9314-9324.1999 |
0.451 |
|
1999 |
Russell DW, Kay MA. Adeno-associated virus vectors and hematology Blood. 94: 864-874. PMID 10419876 DOI: 10.1182/Blood.V94.3.864.415K34_864_874 |
0.449 |
|
1999 |
Nakai H, Iwaki Y, Kay MA, Couto LB. Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver Journal of Virology. 73: 5438-5447. PMID 10364291 DOI: 10.1128/Jvi.73.7.5438-5447.1999 |
0.507 |
|
1999 |
Patijn GA, Kay MA. Hepatic gene therapy using adeno-associated virus vectors. Seminars in Liver Disease. 19: 61-69. PMID 10349684 DOI: 10.1055/S-2007-1007098 |
0.454 |
|
1999 |
Schowalter DB, Himeda CL, Winther BL, Wilson CB, Kay MA. Implication of interfering antibody formation and apoptosis as two different mechanisms leading to variable duration of adenovirus-mediated transgene expression in immune-competent mice Journal of Virology. 73: 4755-4766. PMID 10233936 DOI: 10.1128/Jvi.73.6.4755-4766.1999 |
0.344 |
|
1999 |
Snyder RO, Miao C, Meuse L, Tubb J, Donahue BA, Lin HF, Stafford DW, Patel S, Thompson AR, Nichols T, Read MS, Bellinger DA, Brinkhous KM, Kay MA. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors Nature Medicine. 5: 64-70. PMID 9883841 DOI: 10.1038/4751 |
0.39 |
|
1999 |
Mizuguchi H, Kay MA. Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method. Human Gene Therapy. 9: 2577-83. PMID 9853524 DOI: 10.1089/Hum.1998.9.17-2577 |
0.43 |
|
1998 |
Lee CA, Kessler CM, Varon D, Martinowitz U, Heim M, Kay MA. Hepatic gene therapy for haemophilia B. Haemophilia. 4: 389-392. PMID 9873759 DOI: 10.1046/J.1365-2516.1998.440389.X |
0.452 |
|
1998 |
Patijn GA, Lieber A, Schowalter DB, Schwall R, Kay MA. Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice. Hepatology (Baltimore, Md.). 28: 707-16. PMID 9731563 DOI: 10.1002/Hep.510280317 |
0.469 |
|
1998 |
Wilson CB, Embree LJ, Schowalter D, Albert R, Aruffo A, Hollenbaugh D, Linsley P, Kay MA. Transient inhibition of CD28 and CD40 ligand interactions prolongs adenovirus-mediated transgene expression in the lung and facilitates expression after secondary vector administration. Journal of Virology. 72: 7542-50. PMID 9696851 DOI: 10.1128/Jvi.72.9.7542-7550.1998 |
0.434 |
|
1998 |
Patijn GA, Lieber A, Meuse L, Winther B, Kay MA. High-efficiency retrovirus-mediated gene transfer into the livers of mice. Human Gene Therapy. 9: 1449-56. PMID 9681416 DOI: 10.1089/Hum.1998.9.10-1449 |
0.533 |
|
1998 |
Miao CH, Snyder RO, Schowalter DB, Patijn GA, Donahue B, Winther B, Kay MA. The kinetics of rAAV integration in the liver. Nature Genetics. 19: 13-5. PMID 9590280 DOI: 10.1038/Ng0598-13 |
0.333 |
|
1998 |
Lieber A, He CY, Kay MA. Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo. Nature Biotechnology. 15: 1383-7. PMID 9415891 DOI: 10.1038/Nbt1297-1383 |
0.456 |
|
1998 |
Wilson CB, Embree LJ, Schowalter D, Albert R, Aruffo A, Hollenbaugh D, Linsley P, Kay MA. Transient Inhibition of CD28 and CD40 Ligand Interactions Prolongs Adenovirus-Mediated Transgene Expression in the Lung and Facilitates Expression after Secondary Vector Administration Journal of Virology. 72: 7542-7550. DOI: 10.1128/jvi.72.9.7542-7550.1998 |
0.322 |
|
1998 |
Lieber A, He C, Meuse L, Himeda C, Wilson C, Kay MA. Inhibition of NF-κB Activation in Combination with Bcl-2 Expression Allows for Persistence of First-Generation Adenovirus Vectors in the Mouse Liver Journal of Virology. 72: 9267-9277. DOI: 10.1128/Jvi.72.11.9267-9277.1998 |
0.455 |
|
1998 |
Patijn GA, Lieber A, Sohowalter DB, Schwall R, Terpstra OT, Kay MA. Hepatocyte growth factor (HGF) induces high level hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice European Journal of Gastroenterology & Hepatology. 10: A58. DOI: 10.1097/00042737-199812000-00191 |
0.392 |
|
1997 |
Barr D, Kay MA. Methods for delivery of genes to hepatocytes in vivo using recombinant adenovirus vectors. Methods in Molecular Medicine. 7: 205-212. PMID 24493428 DOI: 10.1385/0-89603-484-4:205 |
0.443 |
|
1997 |
Kay MA, Liu D, Hoogerbrugge PM. Gene therapy Proceedings of the National Academy of Sciences of the United States of America. 94: 12744-12746. PMID 9398067 DOI: 10.1073/Pnas.94.24.12744 |
0.364 |
|
1997 |
Nelson JE, Kay MA. Persistence of recombinant adenovirus in vivo is not dependent on vector DNA replication. Journal of Virology. 71: 8902-7. PMID 9343256 DOI: 10.1128/Jvi.71.11.8902-8907.1997 |
0.487 |
|
1997 |
Schowalter D, Meuse L, Wilson C, Linsley P, Kay M. Constitutive expression of murine CTLA4Ig from a recombinant adenovirus vector results in prolonged transgene expression Gene Therapy. 4: 853-860. PMID 9338015 DOI: 10.1038/Sj.Gt.3300466 |
0.42 |
|
1997 |
Schowalter DB, Kay MA. Gene Therapy: A Status Report Pediatric Annals. 26: 562-568. PMID 9302719 DOI: 10.3928/0090-4481-19970901-12 |
0.349 |
|
1997 |
Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, Nagy D, Gown AM, Winther B, Meuse L, Cohen LK, Thompson AR, Kay MA. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genetics. 16: 270-6. PMID 9207793 DOI: 10.1038/Ng0797-270 |
0.482 |
|
1997 |
Kay MA. Adenoviral Vectors for Hepatic Gene Transfer in Animals Chest. 111. PMID 9184566 DOI: 10.1378/Chest.111.6_Supplement.138S |
0.429 |
|
1997 |
Schowalter D, Tubb J, Liu M, Wilson C, Kay M. Heterologous expression of adenovirus E3-gp19K in an E1a-deleted adenovirus vector inhibits MHC I expression in vitro, but does not prolong transgene expression in vivo Gene Therapy. 4: 351-360. PMID 9176522 DOI: 10.1038/Sj.Gt.3300398 |
0.372 |
|
1997 |
Kay MA, Meuse L, Gown AM, Linsley P, Hollenbaugh D, Aruffo A, Ochs HD, Wilson CB. Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver Proceedings of the National Academy of Sciences of the United States of America. 94: 4686-4691. PMID 9114052 DOI: 10.1073/Pnas.94.9.4686 |
0.463 |
|
1997 |
Vrancken Peeters MJ, Patijn GA, Lieber A, Perkins J, Kay MA. Expansion of donor hepatocytes after recombinant adenovirus-induced liver regeneration in mice. Hepatology (Baltimore, Md.). 25: 884-8. PMID 9096592 DOI: 10.1002/Hep.510250416 |
0.389 |
|
1997 |
Kay MA, Fausto N. Liver regeneration: prospects for therapy based on new technologies. Molecular Medicine Today. 3: 108-15. PMID 9095485 DOI: 10.1016/S1357-4310(96)10062-9 |
0.377 |
|
1997 |
Overturf K, al-Dhalimy M, Ou CN, Finegold M, Tanguay R, Lieber A, Kay M, Grompe M. Adenovirus-mediated gene therapy in a mouse model of hereditary tyrosinemia type I. Human Gene Therapy. 8: 513-21. PMID 9095403 DOI: 10.1089/Hum.1997.8.5-513 |
0.429 |
|
1997 |
Thompson AR, Kay MA. Nonviral gene transfer to the liver. Hepatology (Baltimore, Md.). 24: 1541-2. PMID 8938194 DOI: 10.1002/Hep.510240641 |
0.395 |
|
1997 |
Peeters MJ, Patijn GA, Lieber A, Meuse L, Kay MA. Adenovirus-mediated hepatic gene transfer in mice: comparison of intravascular and biliary administration. Human Gene Therapy. 7: 1693-9. PMID 8886840 DOI: 10.1089/Hum.1996.7.14-1693 |
0.513 |
|
1997 |
Lieber A, He CY, Meuse L, Schowalter D, Kirillova I, Winther B, Kay MA. The role of Kupffer cell activation and viral gene expression in early liver toxicity after infusion of recombinant adenovirus vectors. Journal of Virology. 71: 8798-8807. DOI: 10.1128/Jvi.71.11.8798-8807.1997 |
0.49 |
|
1996 |
Murry CE, Kay MA, Bartosek T, Hauschka SD, Schwartz SM. Muscle differentiation during repair of myocardial necrosis in rats via gene transfer with MyoD. The Journal of Clinical Investigation. 98: 2209-17. PMID 8941636 DOI: 10.1172/Jci119030 |
0.341 |
|
1996 |
Vrancken Peeters MJ, Perkins AL, Kay MA. Method for multiple portal vein infusions in mice: quantitation of adenovirus-mediated hepatic gene transfer. Biotechniques. 20: 278-85. PMID 8825158 DOI: 10.2144/96202Rr05 |
0.451 |
|
1996 |
Grim J, Deshane J, Feng M, Lieber A, Kay M, Curiel DT. erbB-2 Knockout Employing an Intracellular Single-chain Antibody (sFv) Accomplishes Specific Toxicity in erbB-2-expressing Lung Cancer Cells American Journal of Respiratory Cell and Molecular Biology. 15: 348-354. PMID 8810638 DOI: 10.1165/Ajrcmb.15.3.8810638 |
0.317 |
|
1996 |
Lieber A, Kay MA. Adenovirus-mediated expression of ribozymes in mice. Journal of Virology. 70: 3153-3158. DOI: 10.1128/Jvi.70.5.3153-3158.1996 |
0.487 |
|
1996 |
Liu ML, Winther BL, Kay MA. Pseudotransduction of hepatocytes by using concentrated pseudotyped vesicular stomatitis virus G glycoprotein (VSV-G)-Moloney murine leukemia virus-derived retrovirus vectors: comparison of VSV-G and amphotropic vectors for hepatic gene transfer. Journal of Virology. 70: 2497-2502. DOI: 10.1128/Jvi.70.4.2497-2502.1996 |
0.471 |
|
1996 |
Lieber A, He CY, Kirillova I, Kay MA. Recombinant adenoviruses with large deletions generated by Cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo. Journal of Virology. 70: 8944-8960. DOI: 10.1128/Jvi.70.12.8944-8960.1996 |
0.521 |
|
1996 |
Lieber A, He CY, Polyak SJ, Gretch DR, Barr D, Kay MA. Elimination of hepatitis C virus RNA in infected human hepatocytes by adenovirus-mediated expression of ribozymes. Journal of Virology. 70: 8782-8791. DOI: 10.1128/Jvi.70.12.8782-8791.1996 |
0.366 |
|
1995 |
Kay MA. Hepatic gene therapy for hemophilia B. Advances in Experimental Medicine and Biology. 386: 229-234. PMID 8851032 DOI: 10.1007/978-1-4613-0331-2_22 |
0.458 |
|
1995 |
Deshane J, Siegal GP, Alvarez RD, Wang MH, Feng M, Cabrera G, Liu T, Kay M, Curiel DT. Targeted tumor killing via an intracellular antibody against erbB-2 Journal of Clinical Investigation. 96: 2980-2989. PMID 8675670 DOI: 10.1172/Jci118370 |
0.329 |
|
1995 |
Kay MA, Graham F, Leland F, Woo SL. Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology (Baltimore, Md.). 21: 815-9. PMID 7875680 DOI: 10.1002/Hep.1840210331 |
0.59 |
|
1995 |
Lieber A, Vrancken Peeters MJ, Kay MA. Adenovirus-mediated transfer of the amphotropic retrovirus receptor cDNA increases retroviral transduction in cultured cells. Human Gene Therapy. 6: 5-11. PMID 7703287 DOI: 10.1089/Hum.1995.6.1-5 |
0.44 |
|
1995 |
Lieber A, Vrancken Peeters MJ, Meuse L, Fausto N, Perkins J, Kay MA. Adenovirus-mediated urokinase gene transfer induces liver regeneration and allows for efficient retrovirus transduction of hepatocytes in vivo. Proceedings of the National Academy of Sciences of the United States of America. 92: 6210-4. PMID 7597103 DOI: 10.1073/Pnas.92.13.6210 |
0.487 |
|
1995 |
Wilson C, Kay MA. Immunomodulation to enhance gene therapy. Nature Medicine. 1: 887-9. PMID 7585212 DOI: 10.1038/Nm0995-887 |
0.358 |
|
1995 |
Fang B, Eisensmith RC, Wang H, Kay MA, Cross RE, Landen CN, Gordon G, Bellinger DA, Read MS, Hu PC. Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression. Human Gene Therapy. 6: 1039-44. PMID 7578416 DOI: 10.1089/Hum.1995.6.8-1039 |
0.45 |
|
1995 |
Lieber A, Peeters MJ, Gown A, Perkins J, Kay MA. A modified urokinase plasminogen activator induces liver regeneration without bleeding. Human Gene Therapy. 6: 1029-37. PMID 7578415 DOI: 10.1089/Hum.1995.6.8-1029 |
0.499 |
|
1995 |
Kay MA, Holterman AX, Meuse L, Gown A, Ochs HD, Linsley PS, Wilson CB. Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration. Nature Genetics. 11: 191-7. PMID 7550348 DOI: 10.1038/Ng1095-191 |
0.495 |
|
1994 |
Kay MA. Hepatocyte transplantation for liver gene therapy. Cell Transplantation. 2: 405-6. PMID 8162281 DOI: 10.1177/096368979300200506 |
0.367 |
|
1994 |
Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wiehle S, Fang B, Bellinger D, Finegold M, Thompson AR. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proceedings of the National Academy of Sciences of the United States of America. 91: 2353-7. PMID 8134398 DOI: 10.1073/Pnas.91.6.2353 |
0.432 |
|
1994 |
Kay MA, Woo SL. Gene therapy for metabolic disorders. Trends in Genetics : Tig. 10: 253-7. PMID 8091506 DOI: 10.1016/0168-9525(94)90173-2 |
0.562 |
|
1994 |
Kay MA, Woo SLC. Gene Therapy for Metabolic Diseases Ilar Journal. 36: 47-53. DOI: 10.1093/Ilar.36.3-4.47 |
0.308 |
|
1994 |
Kolodka TM, Finegold M, Kay MA, Woo SL. Hepatic gene therapy: Efficient retroviral mediated gene transfer into rat hepatocytes in vivo Somatic Cell and Molecular Genetics. 20: 251. DOI: 10.1007/BF02254765 |
0.547 |
|
1993 |
Li Q, Kay MA, Finegold M, Stratford-Perricaudet LD, Woo SL. Assessment of recombinant adenoviral vectors for hepatic gene therapy. Human Gene Therapy. 4: 403-9. PMID 8399487 DOI: 10.1089/Hum.1993.4.4-403 |
0.622 |
|
1993 |
Cristiano RJ, Smith LC, Kay MA, Brinkley BR, Woo SL. Hepatic gene therapy: efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex. Proceedings of the National Academy of Sciences of the United States of America. 90: 11548-52. PMID 8265587 DOI: 10.1073/Pnas.90.24.11548 |
0.629 |
|
1993 |
Kay MA, Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM, Woo SLC. In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs Science. 262: 117. PMID 8211118 DOI: 10.1126/Science.8211118 |
0.468 |
|
1993 |
Kolodka TM, Finegold M, Kay MA, Woo SL. Hepatic gene therapy: efficient retroviral-mediated gene transfer into rat hepatocytes in vivo. Somatic Cell and Molecular Genetics. 19: 491-7. PMID 7980740 DOI: 10.1007/Bf01233254 |
0.603 |
|
1992 |
Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu TJ, Finegold M, Darlington G, Pokorny W, Woo SLC. Expression of human α1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes Proceedings of the National Academy of Sciences of the United States of America. 89: 89-93. PMID 1729724 DOI: 10.1073/Pnas.89.1.89 |
0.41 |
|
1992 |
Kay MA, Ponder KP, Woo SL. Human gene therapy: present and future. Breast Cancer Research and Treatment. 21: 83-93. PMID 1627818 DOI: 10.1007/Bf01836954 |
0.625 |
|
1992 |
Kay MA, Li Q, Liu TJ, Leland F, Toman C, Finegold M, Woo SL. Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Human Gene Therapy. 3: 641-7. PMID 1482704 DOI: 10.1089/Hum.1992.3.6-641 |
0.652 |
|
1992 |
Liu TJ, Kay MA, Darlington GJ, Woo SL. Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice. Somatic Cell and Molecular Genetics. 18: 89-96. PMID 1312261 DOI: 10.1007/Bf01233451 |
0.612 |
|
1988 |
Kay MA, Zhang JY, Jacobs-Lorena M. Identification and germline transformation of the ribosomal protein rp21 gene of Drosophila: Complementation analysis with the Minute QIII locus reveals nonidentity Mgg Molecular & General Genetics. 213: 354-358. PMID 3185506 DOI: 10.1007/Bf00339602 |
0.386 |
|
1987 |
Qian S, Zhang JY, Kay MA, Jacobs-Lorena M. Structural analysis of the Drosophila rpA1 gene, a member of the eucaryotic 'A' type ribosomal protein family Nucleic Acids Research. 15: 987-1003. PMID 3103101 DOI: 10.1093/Nar/15.3.987 |
0.356 |
|
1987 |
Kay MA, Jacobs-Lorena M. Developmental genetics of ribosome synthesis in Drosophila Trends in Genetics. 3: 347-351. DOI: 10.1016/0168-9525(87)90295-2 |
0.355 |
|
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