Mark A. Kay, MD PhD - Publications

Affiliations: 
Stanford University, Palo Alto, CA 

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

Year Citation  Score
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  0.44
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  0.48
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  0.36
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  0.48
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  0.36
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  0.48
2018 Paulk NK, Pekrun K, Charville GW, Maguire-Nguyen K, Wosczyna MN, Xu J, Zhang Y, Lisowski L, Yoo B, Vilches-Moure JG, Lee GK, Shrager JB, Rando TA, Kay MA. Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle. Molecular Therapy. Methods & Clinical Development. 10: 144-155. PMID 30101152 DOI: 10.1016/j.omtm.2018.06.001  0.36
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  0.44
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  0.36
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  0.36
2016 Chak K, Roy-Chaudhuri B, Kim HK, Kemp KC, Porter BE, Kay MA. Increased precursor microRNA-21 following status epilepticus can compete with mature microRNA-21 to alter translation. Experimental Neurology. PMID 27725160 DOI: 10.1016/j.expneurol.2016.10.003  0.44
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  0.44
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  0.48
2015 Kay MA. Selecting the Best AAV Capsid for Human Studies. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 1800-1. PMID 26689120 DOI: 10.1038/mt.2015.206  1
2015 Haussecker D, Kay MA. RNA interference. Drugging RNAi. Science (New York, N.Y.). 347: 1069-70. PMID 25745148 DOI: 10.1126/science.1252967  1
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  1
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  1
2015 Valdmanis PN, Roy-Chaudhuri B, Kim HK, Sayles LC, Zheng Y, Chuang CH, Caswell DR, Chu K, Zhang Y, Winslow MM, Sweet-Cordero EA, Kay MA. Upregulation of the microRNA cluster at the Dlk1-Dio3 locus in lung adenocarcinoma. Oncogene. 34: 94-103. PMID 24317514 DOI: 10.1038/onc.2013.523  0.48
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  1
2014 Roy-Chaudhuri B, Valdmanis PN, Zhang Y, Wang Q, Luo QJ, Kay MA. Regulation of microRNA-mediated gene silencing by microRNA precursors. Nature Structural & Molecular Biology. 21: 825-32. PMID 25086740 DOI: 10.1038/nsmb.2862  1
2014 Wang Y, Liang P, Lan F, Wu H, Lisowski L, Gu M, Hu S, Kay MA, Urnov FD, Shinnawi R, Gold JD, Gepstein L, Wu JC. Genome editing of isogenic human induced pluripotent stem cells recapitulates long QT phenotype for drug testing. Journal of the American College of Cardiology. 64: 451-9. PMID 25082577 DOI: 10.1016/j.jacc.2014.04.057  1
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  1
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  1
2014 Kay MA, Walker BD. Engineering cellular resistance to HIV. The New England Journal of Medicine. 370: 968-9. PMID 24597871 DOI: 10.1056/NEJMe1400593  1
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  1
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  1
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  1
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  1
2013 Tahara H, Kay MA, Yasui W, Tahara E. MicroRNAs in Cancer: the 22nd Hiroshima Cancer Seminar/the 4th Japanese Association for RNA Interference Joint International Symposium, 30 August 2012, Grand Prince Hotel Hiroshima. Japanese Journal of Clinical Oncology. 43: 579-82. PMID 23487440 DOI: 10.1093/jjco/hyt037  1
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  1
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  1
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  1
2012 Gu S, Jin L, Zhang Y, Huang Y, Zhang F, Valdmanis PN, Kay MA. The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo. Cell. 151: 900-11. PMID 23141545 DOI: 10.1016/j.cell.2012.09.042  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
2010 Gu S, Kay MA. How do miRNAs mediate translational repression? Silence. 1: 11. PMID 20459656 DOI: 10.1186/1758-907X-1-11  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
2009 Gu S, Jin L, Zhang F, Sarnow P, Kay MA. Biological basis for restriction of microRNA targets to the 3' untranslated region in mammalian mRNAs. Nature Structural & Molecular Biology. 16: 144-50. PMID 19182800 DOI: 10.1038/nsmb.1552  1
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  1
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  1
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  1
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  1
2008 Rossi J, Zamore P, Kay MA. Wandering eye for RNAi. Nature Medicine. 14: 611. PMID 18535575 DOI: 10.1038/nm0608-611  1
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  1
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  1
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  1
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  1
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  1
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  1
2008 Kay MA. Prime time for small RNA-based therapeutics. Human Gene Therapy. 19: 15-6. PMID 18211227 DOI: 10.1089/hum.2007.1220  1
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  1
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  1
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  1
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  1
2007 Kay MA. AAV vectors and tumorigenicity. Nature Biotechnology. 25: 1111-3. PMID 17921994 DOI: 10.1038/nbt1007-1111  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  0.32
2007 Kay MA. MicroRNAs outwit immune limitations in gene therapy Blood. 110: 4136-4137. DOI: 10.1182/blood-2007-09-111062  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
2005 Ohashi K, Kay MA, Kuge H, Yokoyama T, Kanehiro H, Hisanaga M, Ko S, Nagao M, Sho M, Nakajima Y. Heterotopically transplanted hepatocyte survival depends on extracellular matrix components. Transplantation Proceedings. 37: 4587-8. PMID 16387176 DOI: 10.1016/j.transproceed.2005.11.049  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
2004 Ohashi K, Kay MA. Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice. Transplantation Proceedings. 36: 2469-70. PMID 15561284 DOI: 10.1016/j.transproceed.2004.07.072  1
2004 Ganaha F, Ohashi K, Do YS, Lee J, Sugimoto K, Minamiguchi H, Elkins CJ, Sameni D, Modanlou S, Ali M, Kao EY, Kay MA, Waugh JM, Dake MD. Efficient inhibition of in-stent restenosis by controlled stent-based inhibition of elastase: a pilot study. Journal of Vascular and Interventional Radiology : Jvir. 15: 1287-93. PMID 15525749 DOI: 10.1097/01.RVI.0000141340.67588.4F  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
2003 Kay MA, Nakai H. Looking into the safety of AAV vectors. Nature. 424: 251. PMID 12867952 DOI: 10.1038/424251b  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
2003 McCaffrey AP, Kay MA. RNA interference gets infectious Gene Therapy. 10: 1205. DOI: 10.1038/sj.gt.3302035  1
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  1
2002 McCaffrey AP, Kay MA. A story of mice and men. Gene Therapy. 9: 1563. PMID 12424608 DOI: 10.1038/sj.gt.3301890  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
1999 Vilain E, Merrer MLE, Lecointre C, Desangles F, Kay MA, Maroteaux P, Mccabe ERB. IMAGe, a new clinical association of Intrauterine growth retardation, Metaphyseal dysplasia, adrenal hypoplasia congenita, and Genital anomalies Journal of Clinical Endocrinology and Metabolism. 84: 4335-4340. PMID 10599684  1
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  1
1999 Zen K, Karsan A, Stempien-Otero A, Yee E, Tupper J, Li X, Eunson T, Kay MA, Wilson CB, Winn RK, Harlan JM. NF-kappaB activation is required for human endothelial survival during exposure to tumor necrosis factor-alpha but not to interleukin-1beta or lipopolysaccharide. The Journal of Biological Chemistry. 274: 28808-15. PMID 10497254 DOI: 10.1074/jbc.274.40.28808  1
1999 Kay MA, High K. Gene therapy for the hemophilias Proceedings of the National Academy of Sciences of the United States of America. 96: 9973-9975. PMID 10468539 DOI: 10.1073/pnas.96.18.9973  1
1999 Mizuguchi H, Kay MA. A simple method for constructing E1- and E1/E4-deleted recombinant adenoviral vectors Human Gene Therapy. 10: 2013-2017. PMID 10466635 DOI: 10.1089/10430349950017374  1
1999 Russell DW, Kay MA. Adeno-associated virus vectors and hematology Blood. 94: 864-874. PMID 10419876  1
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  1
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  1
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  1
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  1
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  1
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  1
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  1
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  0.44
1994 Kay MA, Woo SL. Gene therapy for metabolic disorders. Trends in Genetics : Tig. 10: 253-7. PMID 8091506  1
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  1
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  1
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  1
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  1
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  1
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  1
1992 Kay MA, Ponder KP, Woo SL. Human gene therapy: present and future. Breast Cancer Research and Treatment. 21: 83-93. PMID 1627818  1
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  1
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  1
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  1
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  1
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  1
1985 Kay MA, Jacobs-Lorena M. Selective translational regulation of ribosomal protein gene expression during early development of Drosophila melanogaster Molecular and Cellular Biology. 5: 3583-3592. PMID 3939320 DOI: 10.1128/mcb.5.12.3583  1
Show low-probability matches.