KAY LAB PUBLICATIONS





2018

262. 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. 2018; 10:144-155. [PDF]

261. 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, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA. 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. 2018; 26(1):289-303. [PDF]

2017

260. 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. 2017; 8(1):2053. [PDF]

259. 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. A transfer-RNA-derived small RNA regulates ribosome biogenesis. Nature. 2017; 552(7683):57-62. [PDF]

258. 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, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. Science translational medicine. 2017; 9(418). [PDF]

257. 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. 2017; 25(11):2477-2489. [PDF]

256. 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. 2017; 9(10):1346-1355. [PDF]

255. 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. 2017; 25(5):1187-1198. [PDF]

254. Valdmanis PN, Kay MA. Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond. Human gene therapy. 2017; 28(4):361-372. [PDF]

253. 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. 2017; 45(1):311-326. [PDF]

252. Lu J, Williams JA, Luke J, Zhang F, Chu K, Kay MA. A 5' Noncoding Exon Containing Engineered Intron Enhances Transgene Expression from Recombinant AAV Vectors in vivo. Human gene therapy. 2017; 28(1):125-134. [PDF]

2016

251. 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. 2016; 286:137-146. [PDF]

250. Disterer P, Kay MA, Parker GC. Dieter C. Gruenert, PhD (1949-2016). Nucleic acid therapeutics. 2016; 26(4):266-7. [PDF]

249. Chu J, Oh Y, Sens A, Ataie N, Dana H, Macklin JJ, Laviv T, Welf ES, Dean KM, Zhang F, Kim BB, Tang CT, Hu M, Baird MA, Davidson MW, Kay MA, Fiolka R, Yasuda R, Kim DS, Ng HL, Lin MZ. A bright cyan-excitable orange fluorescent protein facilitates dual-emission microscopy and enhances bioluminescence imaging in vivo. Nature biotechnology. 2016; 34(7):760-7. [PDF]

248. 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. 2016; 8(342):342ra79. [PDF]

247. 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. 2016; 22(5):557-62. [PDF]

246. Srivastava A, Kay MA, Athanasopoulos T, Angastiniotis M, Anagnostopoulos A, Karponi G, Yannaki E, Zon LI, Lederer CW, Phylactides MS, Kleanthous M. A Tribute to George Stamatoyannopoulos. Human gene therapy. 2016; 27(4):280-6. [PDF]

2015

245. Kay MA. Selecting the Best AAV Capsid for Human Studies. Molecular therapy : the journal of the American Society of Gene Therapy. 2015; 23(12):1800-1. [PDF]

244. Mellins ED, Kay MA. Viral Vectors Take On HIV Infection. The New England journal of medicine. 2015; 373(8):770-2. [PDF]

243. Haussecker D, Kay MA. RNA interference. Drugging RNAi. Science (New York, N.Y.). 2015; 347(6226):1069-70. [PDF]

242. 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. 2015; 26(1):5-14. [PDF]

241. 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. 2015; 5:8081. [PDF]

240. 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. 2015; 517(7534):360-4. [PDF]

239. 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. 2015; 34(1):94-103. [PDF]

2014

238. 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. 2014; [PDF]

237. Sebastiano V, Zhen HH, Haddad B, Bashkirova E, Melo SP, Wang P, Leung TL, Siprashvili Z, Tichy A, Li J, Ameen M, Hawkins J, Lee S, Li L, Schwertschkow A, Bauer G, Lisowski L, Kay MA, Kim SK, Lane AT, Wernig M, Oro AE. Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa. Science translational medicine. 2014; 6(264):264ra163. [PDF]

236. 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, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. The New England journal of medicine. 2014; 371(21):1994-2004. [PDF]

235. 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. 2014; 42(19):12169-76. [PDF]

234. 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. 2014; 21(9):825-32. [PDF]

233. 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. 2014; 64(5):451-9. [PDF]

232. 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. 2014; 8(2):371-81. [PDF]

231. Phillips N, Kay MA. Characterization of vector-based delivery of neurogenin-3 in murine diabetes. Human gene therapy. 2014; 25(7):651-61. [PDF]

230. Chung J, Scherer LJ, Gu A, Gardner AM, Torres-Coronado M, Epps EW, Digiusto DL, Rossi JJ. Optimized lentiviral vectors for HIV gene therapy: multiplexed expression of small RNAs and inclusion of MGMT(P140K) drug resistance gene. Molecular therapy : the journal of the American Society of Gene Therapy. 2014; 22(5):952-63. [PDF]

229. 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. 2014; 22(4):692-701. [PDF]

228. 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. 2014; 22(4):725-33. [PDF]

227. Kay MA, Walker BD. Engineering cellular resistance to HIV. The New England journal of medicine. 2014; 370(10):968-9. [PDF]

226. 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. 2014; 506(7488):382-6. [PDF]

2013

225. Valdmanis PN, Kay MA. The expanding repertoire of circular RNAs. Molecular therapy : the journal of the American Society of Gene Therapy. 2013; 21(6):1112-4. [PDF]

224. 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. 2013; 21(5):954-63. [PDF]

223. 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. 2013; 43(5):579-82. [PDF]

222. 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. 2013; 41(6):3688-98. [PDF]

221. 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. 2013; 21(1):131-8. [PDF]

2012

220. 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, Wu JC. Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circulation research. 2012; 111(12):1494-503. [PDF]

219. 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. 2012; 151(4):900-11. [PDF]

218. 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. 2012; 20(11):2014-7. [PDF]

217. 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. 2012; 20(11):2111-9. [PDF]

216. 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. 2012; 20(10):1912-23. [PDF]

215. 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. 2012; 20(10):1902-11. [PDF]

214. Gu S, Jin L, Huang Y, Zhang F, Kay MA. Slicing-independent RISC activation requires the argonaute PAZ domain. Current biology : CB. 2012; 22(16):1536-42. [PDF]

213. 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. 2012; 40(8):3704-13. [PDF]

2011

212. 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, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. The New England journal of medicine. 2011; 365(25):2357-65. [PDF]

211. 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. 2011; 121(12):4850-60. [PDF]

210. 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. 2011; 124(11 Suppl):S46-54. [PDF]

209. 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. 2011; 108(22):9208-13. [PDF]

208. Kay MA. State-of-the-art gene-based therapies: the road ahead. Nature reviews. Genetics. 2011; 12(5):316-28. [PDF]

207. 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. 2011; 19(3):450-60. [PDF]

206. 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. 2011; 6(1):78-88. [PDF]

205. 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. 2011; 20(5):655-67. [PDF]

2010

204. Kay MA, He CY, Chen ZY. A robust system for production of minicircle DNA vectors. Nature biotechnology. 2010; 28(12):1287-9. [PDF]

203. 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. 2010; 18(11):1896-906. [PDF]

202. 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. 2010; 120(9):3106-19. [PDF]

201. 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. 2010; 299(3):E384-93. [PDF]

200. 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. 2010; 3(1):13. [PDF]

199. Gu S, Kay MA. How do miRNAs mediate translational repression? Silence. 2010; 1(1):11. [PDF]

198. 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. 2010; 16 Suppl 3:19-23. [PDF]

197. 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.). 2010; 51(4):1200-8. [PDF]

196. 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.). 2010; 16(4):673-95. [PDF]

195. 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. 2010; 7(3):197-9. NIHMSID: NIHMS208396 [PDF]

194. 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. 2010; 18(2):240-2. [PDF]

193. 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. 2010; 18(1):161-70. [PDF]

2009

192. 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.). 2009; 15(11):1971-9. [PDF]

191. 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. 2009; 120(11 Suppl):S230-7. [PDF]

190. 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. 2009; 16(2):144-50. [PDF]

189. 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. 2009; 53(1):18-23. [PDF]

188. 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. 2009; 4(4):547-64. [PDF]

2008

187. 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. 2008; 16(9):1630-6. [PDF]

186. 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. 2008; 283(32):22186-92. [PDF]

185. 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. 2008; 19(8):820-6. [PDF]

184. 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. 2008; 15(7):714-21. [PDF]

183. Rossi J, Zamore P, Kay MA. Wandering eye for RNAi. Nature medicine. 2008; 14(6):611. [PDF]

182. 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. 2008; 82(12):5887-911. [PDF]

181. Haussecker D. The business of RNAi therapeutics. Human gene therapy. 2008; 19(5):451-62. [PDF]

180. 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. 2008; 19(5):429-30. [PDF]

179. 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. 2008; 16(5):931-41. [PDF]

178. 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. 2008; 16(3):548-56. [PDF]

177. Lazarus JJ, Kay MA, McCarter AL, Wooten RM. Viable Borrelia burgdorferi enhances interleukin-10 production and suppresses activation of murine macrophages. Infection and immunity. 2008; 76(3):1153-62. [PDF]

176. 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.). 2008; 47(2):706-18. [PDF]

175. Kay MA. Prime time for small RNA-based therapeutics. Human gene therapy. 2008; 19(1):15-6. [PDF]

2007

174. Grimm D, Kay MA. Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? The Journal of clinical investigation. 2007; 117(12):3633-41. [PDF]

173. Garrison BS, Yant SR, Mikkelsen JG, Kay MA. Postintegrative gene silencing within the Sleeping Beauty transposition system. Molecular and cellular biology. 2007; 27(24):8824-33. [PDF]

172. 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. 2007; 18(12):1193-204. [PDF]

171. 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. 2007; 15(11):1955-62. [PDF]

170. 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. 2007; 18(10):871-80. [PDF]

169. Kay MA. AAV vectors and tumorigenicity. Nature biotechnology. 2007; 25(10):1111-3. [PDF]

168. 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. 2007; 81(20):11290-303. [PDF]

167. 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. 2007; 81(20):11304-21. [PDF]

166. 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. 2007; 104(37):14771-6. [PDF]

165. 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. 2007; 25(8):903-10. [PDF]

164. 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. 2007; 15(7):1273-9. [PDF]

163. 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. 2007; 15(7):1348-55. [PDF]

162. 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. 2007; 15(5):878-88. [PDF]

161. Yant SR, Huang Y, Akache B, Kay MA. Site-directed transposon integration in human cells. Nucleic acids research. 2007; 35(7):e50. [PDF]

160. 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, Hirsch B, Yant SR, Kay MA, Peister A, Prockop DJ, Fibbe WE, Blazar BR. Sarcoma derived from cultured mesenchymal stem cells. Stem cells (Dayton, Ohio). 2007; 25(2):371-9. [PDF]

159. 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. 2007; 15(2):255-63. [PDF]

158. 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. 2007; 15(2):330-9. [PDF]

157. 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. 2007; 15(1):146-56. [PDF]

156. Grimm D, Kay MA. RNAi and gene therapy: a mutual attraction. Hematology. American Society of Hematology. Education Program. 2007; :473-81. [PDF]

2006

155. 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. 2006; 17(11):1077-94. [PDF]

154. 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. 2006; 80(19):9831-6. [PDF]

153. 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. 2006; 14(1):45-53. [PDF]

152. 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. 2006; 441(7092):537-41. [PDF]

151. 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. 2006; 107(10):4182-8. [PDF]

150. Grimm D, Kay MA. Therapeutic short hairpin RNA expression in the liver: viral targets and vectors. Gene therapy. 2006; 13(6):563-75. [PDF]

149. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature medicine. 2006; 12(3):342-7. [PDF]

148. 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. 2006; 80(1):426-39. [PDF]

2005

147. 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. 2005; 37(10):4587-8. [PDF]

146. Ehrhardt A, Kay MA. Gutted adenovirus: a rising star on the horizon? Gene therapy. 2005; 12(21):1540-1. [PDF]

145. 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. 2005; 12(1):42-8. [PDF]

144. 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. 2005; 11(5):695-706. [PDF]

143. 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. 2005; 16(5):558-70. [PDF]

142. 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. 2005; 79(6):3606-14. [PDF]

141. 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. 2005; 25(6):2085-94. [PDF]

140. 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. 2005; 16(3):299-306. [PDF]

139. 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. 2005; 79(1):214-24. [PDF]

138. 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.). 2005; 41(1):132-40. [PDF]

137. Grimm D, Pandey K, Kay MA. Adeno-associated virus vectors for short hairpin RNA expression. Methods in enzymology. 2005; 392:381-405. [PDF]

136. 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. 2005; 16(1):126-31. [PDF]

135. 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. 2005; 14(9):621-7. [PDF]

134. 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. 2005; 70:217-24. [PDF]

2004

133. 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. 2004; 15(11):1287-93. [PDF]

132. Ohashi K, Kay MA. Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice. Transplantation proceedings. 2004; 36(8):2469-70. [PDF]

131. 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. 2004; 24(20):9239-47. [PDF]

130. 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. 2004; 78(4):530-6. [PDF]

129. Layzer JM, McCaffrey AP, Tanner AK, Huang Z, Kay MA, Sullenger BA. In vivo activity of nuclease-resistant siRNAs. RNA (New York, N.Y.). 2004; 10(5):766-71. [PDF]

128. Chen ZY, He CY, Meuse L, Kay MA. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene therapy. 2004; 11(10):856-64. [PDF]

127. 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. 2004; 15(4):405-13. [PDF]

126. 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. 2004; 78(6):3110-22. [PDF]

2003

125. Yant SR, Kay MA. Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells. Molecular and cellular biology. 2003; 23(23):8505-18. [PDF]

124. 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. 2003; 8(4):654-65. [PDF]

123. 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. 2003; 102(7):2412-9. [PDF]

122. 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. 2003; 102(7):2403-11. [PDF]

121. 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. 2003; 8(3):495-500. [PDF]

120. Chyung YH, Peng PD, Kay MA. System for simultaneous tissue-specific and disease-specific regulation of therapeutic gene expression. Human gene therapy. 2003; 14(13):1255-64. [PDF]

119. 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. 2003; 3(4):281-304. [PDF]

118. 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.). 2003; 38(2):503-8. [PDF]

117. 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. 2003; 112(3):407-14. [PDF]

116. 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. 2003; 8(2):314-23. [PDF]

115. Kay MA, Nakai H. Looking into the safety of AAV vectors. Nature. 2003; 424(6946):251. [PDF]

114. 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. 2003; 34(3):297-302. [PDF]

113. Ehrhardt A, Xu H, Kay MA. Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo. Journal of virology. 2003; 77(13):7689-95. [PDF]

112. 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. 2003; 10(13):1099-104. [PDF]

111. 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. 2003; 14(9):871-81. [PDF]

110. 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. 2003; 7(6):839-50. [PDF]

109. 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. 2003; 21(6):639-44. [PDF]

108. Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nature reviews. Genetics. 2003; 4(5):346-58. [PDF]

107. 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, Skarsgard E, Flake AW, Kay MA, High KA, Glader B. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood. 2003; 101(8):2963-72. [PDF]

106. McCaffrey A, Kay MA, Contag CH. Advancing molecular therapies through in vivo bioluminescent imaging. Molecular imaging. 2003; 2(2):75-86. [PDF]

105. 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. 2003; 14(3):215-25. [PDF]

104. 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. 2003; 19(1):144-51. [PDF]

103. 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 : the journal of the American Society of Gene Therapy. 2003; 7(1):101-11. [PDF]

102. 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 : the journal of the American Society of Gene Therapy. 2003; 7(1):112-21. [PDF]

2002

101. McCaffrey AP, Kay MA. A story of mice and men. Gene therapy. 2002; 9(23):1563. [PDF]

100. 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. 2002; 6(6):759-69. [PDF]

99. 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. 2002; 20(11):1124-8. [PDF]

98. 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. 2002; 76(22):11343-9. [PDF]

97. 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. 2002; 76(20):10465-72. [PDF]

96. 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. 2002; 20(10):999-1005. [PDF]

95. McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. RNA interference in adult mice. Nature. 2002; 418(6893):38-9. [PDF]

94. 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. 2002; 99(11):3923-30. [PDF]

93. 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. 2002; 5(6):676-84. [PDF]

92. Ohashi K, Park F, Kay MA. Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo. Human gene therapy. 2002; 13(5):653-63. [PDF]

2001

91. 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, High KA. 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. 2001; 4(6):586-92. [PDF]

90. Ohashi K, Park F, Kay MA. Hepatocyte transplantation: clinical and experimental application. Journal of molecular medicine (Berlin, Germany). 2001; 79(11):617-30. [PDF]

89. 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. 2001; 98(22):12584-9. [PDF]

88. 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 : the journal of the American Society of Gene Therapy. 2001; 4(3):164-73. [PDF]

87. 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. 2001; 75(15):6969-76. [PDF]

86. Stoll SM, Sclimenti CR, Baba EJ, Meuse L, Kay MA, Calos MP. Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice. Molecular therapy : the journal of the American Society of Gene Therapy. 2001; 4(2):122-9. [PDF]

85. 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. 2001; 30(5):1112-4, 1116. [PDF]

84. 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. 2001; 8(9):730-5. [PDF]

83. 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 : the journal of the American Society of Gene Therapy. 2001; 3(3):403-10. [PDF]

82. Ohashi K, Meuse L, Schwall R, Kay MA. cMet activation allows persistent engraftment of ectopically transplanted xenogenic human hepatocytes in mice. Transplantation proceedings. 2001; 33(1-2):587-8. [PDF]

81. Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nature medicine. 2001; 7(1):33-40. [PDF]

2000

80. 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. 2000; 74(20):9451-63. [PDF]

79. Park F, Ohashi K, Kay MA. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood. 2000; 96(3):1173-6. [PDF]

78. 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. 2000; 1(6):522-32. [PDF]

77. 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. 2000; 18(5):527-32. [PDF]

76. 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. 2000; 25(1):35-41. [PDF]

75. 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. 2000; 74(8):3793-803. [PDF]

74. 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, High KA. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature genetics. 2000; 24(3):257-61. [PDF]

73. 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. 2000; 6(3):327-31. [PDF]

72. Park F, Ohashi K, Chiu W, Naldini L, Kay MA. Efficient lentiviral transduction of liver requires cell cycling in vivo. Nature genetics. 2000; 24(1):49-52. [PDF]

71. 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. 2000; 74(2):721-34. [PDF]

1999

70. Vilain E, Le Merrer M, Lecointre C, Desangles F, Kay MA, Maroteaux P, McCabe ER. IMAGe, a new clinical association of intrauterine growth retardation, metaphyseal dysplasia, adrenal hypoplasia congenita, and genital anomalies. The Journal of clinical endocrinology and metabolism. 1999; 84(12):4335-40. [PDF]

69. Lieber A, Steinwaerder DS, Carlson CA, Kay MA. Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes. Journal of virology. 1999; 73(11):9314-24. [PDF]

68. 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. 1999; 274(40):28808-15. [PDF]

67. Kay MA, High K. Gene therapy for the hemophilias. Proceedings of the National Academy of Sciences of the United States of America. 1999; 96(18):9973-5. [PDF]

66. Mizuguchi H, Kay MA. A simple method for constructing E1- and E1/E4-deleted recombinant adenoviral vectors. Human gene therapy. 1999; 10(12):2013-7. [PDF]

65. Russell DW, Kay MA. Adeno-associated virus vectors and hematology. Blood. 1999; 94(3):864-74. NIHMSID: NIHMS474817 [PDF]

64. Nakai H, Iwaki Y, Kay MA, Couto LB. Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. Journal of virology. 1999; 73(7):5438-47. [PDF]

63. 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. 1999; 73(6):4755-66. [PDF]

62. Patijn GA, Kay MA. Hepatic gene therapy using adeno-associated virus vectors. Seminars in liver disease. 1999; 19(1):61-9. [PDF]

61. 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. 1999; 5(1):64-70. [PDF]

1998

60. Mizuguchi H, Kay MA. Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method. Human gene therapy. 1998; 9(17):2577-83. [PDF]

59. Lieber A, He CY, Meuse L, Himeda C, Wilson C, Kay MA. Inhibition of NF-kappaB activation in combination with bcl-2 expression allows for persistence of first-generation adenovirus vectors in the mouse liver. Journal of virology. 1998; 72(11):9267-77. [PDF]

58. 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. 1998; 72(9):7542-50. [PDF]

57. 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.). 1998; 28(3):707-16. [PDF]

56. Patijn GA, Terpstra OT, Kay MA. Method for continuous infusion into the portal vein of mice. Laboratory animal science. 1998; 48(4):379-83. [PDF]

55. Kay MA. Hepatic gene therapy for haemophilia B. Haemophilia : the official journal of the World Federation of Hemophilia. 1998; 4(4):389-92. [PDF]

54. Patijn GA, Lieber A, Meuse L, Winther B, Kay MA. High-efficiency retrovirus-mediated gene transfer into the livers of mice. Human gene therapy. 1998; 9(10):1449-56. [PDF]

53. Miao CH, Snyder RO, Schowalter DB, Patijn GA, Donahue B, Winther B, Kay MA. The kinetics of rAAV integration in the liver. Nature genetics. 1998; 19(1):13-5. [PDF]

1997

52. Bennett RL, Karayiorgou M, Sobin CA, Norwood TH, Kay MA. Identification of an interstitial deletion in an adult female with schizophrenia, mental retardation, and dysmorphic features: further support for a putative schizophrenia-susceptibility locus at 5q21-23.1. American journal of human genetics. 1997; 61(6):1450-4. [PDF]

51. Lieber A, He CY, Kay MA. Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo. Nature biotechnology. 1997; 15(13):1383-7. [PDF]

50. Kay MA, Liu D, Hoogerbrugge PM. Gene therapy. Proceedings of the National Academy of Sciences of the United States of America. 1997; 94(24):12744-6. [PDF]

49. 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. 1997; 71(11):8798-807. [PDF]

48. Nelson JE, Kay MA. Persistence of recombinant adenovirus in vivo is not dependent on vector DNA replication. Journal of virology. 1997; 71(11):8902-7. [PDF]

47. Schowalter DB, Kay MA. Gene therapy: a status report. Pediatric annals. 1997; 26(9):562-8. [PDF]

46. Schowalter DB, Meuse L, Wilson CB, Linsley PS, Kay MA. Constitutive expression of murine CTLA4Ig from a recombinant adenovirus vector results in prolonged transgene expression. Gene therapy. 1997; 4(8):853-60. [PDF]

45. 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. 1997; 16(3):270-6. [PDF]

44. Kay MA. Adenoviral vectors for hepatic gene transfer in animals. Chest. 1997; 111(6 Suppl):138S-142S. [PDF]

43. 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. 1997; 94(9):4686-91. [PDF]

42. 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.). 1997; 25(4):884-8. [PDF]

41. Schowalter DB, Tubb JC, Liu M, Wilson CB, Kay MA. 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. 1997; 4(4):351-60. [PDF]

40. Bethune C, Bui T, Liu ML, Kay MA, Ho RJ. Development of a high-performance liquid chromatographic assay for G418 sulfate (Geneticin). Antimicrobial agents and chemotherapy. 1997; 41(3):661-4. [PDF]

39. Kay MA, Fausto N. Liver regeneration: prospects for therapy based on new technologies. Molecular medicine today. 1997; 3(3):108-15. [PDF]

38. Barr D, Kay MA. Robbins P, editor. Totowa NJ: Humana Press.Methods for delivery of genes to hepatocytes in vivo using recombinant adenoviral vectors. Gene Therapy Protocols. 1997. [PDF]

37. Brand K, Arnold W, Bartels T, Lieber A, Kay MA, Strauss M, Dörken B. Liver-associated toxicity of the HSV-tk/GCV approach and adenoviral vectors. Cancer gene therapy. 1997; 4(1):9-16. [PDF]

36. Barr D, Kay MA. Methods for delivery of genes to hepatocytes in vivo using recombinant adenovirus vectors. Methods in molecular medicine. 1997; 7:205-12. [PDF]

1996

35. Thompson AR, Kay MA. Nonviral gene transfer to the liver. Hepatology (Baltimore, Md.). 1996; 24(6):1541-2. [PDF]

34. 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. 1996; 70(12):8782-91. [PDF]

33. 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. 1996; 70(12):8944-60. [PDF]

32. 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. 1996; 98(10):2209-17. [PDF]

31. Sandig V, Löser P, Lieber A, Kay MA, Strauss M. HBV-derived promoters direct liver-specific expression of an adenovirally transduced LDL receptor gene. Gene therapy. 1996; 3(11):1002-9. [PDF]

30. 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. 1996; 7(14):1693-9. [PDF]

29. Lieber A, Kay MA. Adenovirus-mediated expression of ribozymes in mice. Journal of virology. 1996; 70(5):3153-8. [PDF]

28. 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. 1996; 70(4):2497-502. [PDF]

27. Vrancken Peeters MJ, Perkins AL, Kay MA. Method for multiple portal vein infusions in mice: quantitation of adenovirus-mediated hepatic gene transfer. BioTechniques. 1996; 20(2):278-85. [PDF]

1995

26. 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. 1995; 11(2):191-7. [PDF]

25. Wilson C, Kay MA. Immunomodulation to enhance gene therapy. Nature medicine. 1995; 1(9):887-9. [PDF]

24. Lieber A, Peeters MJ, Gown A, Perkins J, Kay MA. A modified urokinase plasminogen activator induces liver regeneration without bleeding. Human gene therapy. 1995; 6(8):1029-37. [PDF]

23. 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. 1995; 6(8):1039-44. [PDF]

22. 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. 1995; 92(13):6210-4. [PDF]

21. Scaria A, Curiel DT, Kay MA. Complementation of a human adenovirus early region 4 deletion mutant in 293 cells using adenovirus-polylysine-DNA complexes. Gene therapy. 1995; 2(4):295-8. [PDF]

20. Barr D, Tubb J, Ferguson D, Scaria A, Lieber A, Wilson C, Perkins J, Kay MA. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. Gene therapy. 1995; 2(2):151-5. [PDF]

19. 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.). 1995; 21(3):815-9. [PDF]

18. 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. 1995; 6(1):5-11. [PDF]

17. Kay MA. Hepatic gene therapy for hemophilia B. Advances in experimental medicine and biology. 1995; 386:229-34. [PDF]

1994

16. Kay MA, Woo SL. Gene therapy for metabolic disorders. Trends in genetics : TIG. 1994; 10(7):253-7. [PDF]

15. 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. 1994; 91(6):2353-7. [PDF]

1993

14. 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. 1993; 90(24):11548-52. [PDF]

13. Kay MA, Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science (New York, N.Y.). 1993; 262(5130):117-9. [PDF]

12. Kay MA. Hepatocyte transplantation for liver gene therapy. Cell transplantation. 1993; 2(5):405-6. [PDF]

11. Li Q, Kay MA, Finegold M, Stratford-Perricaudet LD, Woo SL. Assessment of recombinant adenoviral vectors for hepatic gene therapy. Human gene therapy. 1993; 4(4):403-9. [PDF]

1992

10. 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. 1992; 3(6):641-7. [PDF]

9. Liu TJ, Kay MA, Darlington GJ, Woo SL. Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice. Somatic cell and molecular genetics. 1992; 18(1):89-96. [PDF]

8. Kay MA, Ponder KP, Woo SL. Human gene therapy: present and future. Breast cancer research and treatment. 1992; 21(2):83-93. [PDF]

7. Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu T, Finegold M, Darlington G, Pokorny W. Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proceedings of the National Academy of Sciences of the United States of America. 1992; 89(1):89-93. [PDF]

1990

6. Kay MA, McCabe ED. Escherichia coli sepsis and prolonged hypophosphatemia following exertional heat stroke. Pediatrics. 1990; 86(2):307-9. [PDF]

5. Kay MA, O'Brien W, Kessler B, McVie R, Ursin S, Dietrich K, McCabe ER. Transient organic aciduria and methemoglobinemia with acute gastroenteritis. Pediatrics. 1990; 85(4):589-92. [PDF]

1988

4. 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. Molecular & general genetics : MGG. 1988; 213(2-3):354-8. [PDF]

1987

3. 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. 1987; 15(3):987-1003. [PDF]

2. Kay MA, Jacobs-Lorena M. Developmental genetics of ribosome synthesis in Drosophilai. Trends in genetics. 1987; 3:347-351. [PDF]

1985

1. Kay MA, Jacobs-Lorena M. Selective translational regulation of ribosomal protein gene expression during early development of Drosophila melanogaster. Molecular and cellular biology. 1985; 5(12):3583-92. [PDF]