Complete List of Publications in PubMed


Aaron KA, Pekrun K, Atkinson PJ, Billings SE, Abitbol JM, Lee IA, Eltawil Y, Chen YS, Dong W, Nelson RF, Kay MA, Cheng AG. Selection of viral capsids and promoters Mol Ther Methods Clin Dev 2023 Aug 11;30:413-428

Holmes AD, Chan PP, Chen Q, Ivanov P, Drouard L, Polacek N, Kay MA, Lowe TM.  A standardized ontology for naming tRNA-derived RNAs based on molecular origin. Nat Methods 2023 May;20(5):627-628.

Gonzalez-Sandoval A, Pekrun K, Tsuji S, Zhang F, Hung KL, Chang HY, Kay MA.  The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner. Nat Commun 2023 Apr 28;14(1):2448.

Davis-Gardner ME, Weber JA, Xie J, Pekrun K, Alexander EA, Weisgrau KL, Furlott JR, Rakasz EG, Kay MA, Gao G, Farzan M, Gardner MR.  A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors. Front Immunol 2023 Apr 21;14:1105617.

Puzzo F, Zhang C, Powell Gray B, Zhang F, Sullenger BA, Kay MA.  Aptamer-programmable adeno-associated viral vectors as a novel platform for cell-specific gene transfer. Mol Ther Nucleic Acids 2023 Jan 21;31:383-397.


Song R, Pekrun K, Khan TA, Zhang F, Pasca SP, Kay MA. Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model. Mol Ther Methods Clin Dev 2022 September 7; 27:P73-88.

Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson GL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J.  Evaluating the state of the science for adeno-associated virus integratio: An integrated perspective. American Society of Gene and Cell Therapy (ASGCT) Working Group on AAV Integration. Mol Ther 2022 30(8):2646-2663.

Tsuji S, Stephens CJ, Bortuolussi G, Zhang F, Baj B, Jang H, de Alencastro G, Muro AF, Pekrun K, Kay MA. Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice. Nature Biotechnol 2022 40(8):1285-1294.

Lisak M, De Caneva A, Marais T, Barbon E, Biferi MG, Porro F, Barzel A, Zentilin L, Kay MA, Mingozzi F, Muro AF. Promoterless gene targeting approach combined to CRISPR/Cas9 efficiently corrects hemophilia B phenotype in neonatal mice. Front Genome Ed 2022 Mar 11;4:785698.

Than PA, Davis CR, Rennert RC, Morrison SD, Findlay MW, Kay MA, Gurtner GC. Selective microvascular tissue transfection using minicircle DNA for systemic delivery of human coagulation factor IX in a rat model using a therapeutic flap. Plast Reconstr Surg 2022 149(1):117-129.   

Spector LP, Tiffany M, Ferraro NM, Abell NS, Montgomery SB, Kay MA. Evaluating the genomic parameters governing rAAV-mediated homologous recombination. Mol Ther 2021 29(3):1028-1046.   

de Alencastro G, Puzzo F, Pavel-Dinu M, Zhang F, Pillay S, Majzoub K, Tiffany M, Jang H, Sheikali A, Cromer MK, Meetei R, Carette JE, Porteus MH, Pekrun K, Kay MA. Improved genome editing through inhibition of FANCM and members of the BTR Dissolvase Complex. Mol Ther 2021 29(3): 1016-1027.    

Chandler RJ, Venturoni LE, Liao J, Hubbard BT, Schneller JL, Hoffmann V, Gordo S, Zang S, Ko CW, Chau N, Chiang K, Kay MA, Barzel A, Venditti CP. Promoterless, nuclease-free genome editing confers a growth advantage for corrected hepatocytes in mice with methylmalonic acidemia. Hepatology 2021 73(6):2223-2237.    

Luo QJ, Zhang J, Li P, Wang Q, Zhang Y, Roy-Chaudhuri B, Xu J, Kay MA, Zhang QC. RNA structure probing reveals the structural basis of dicer binding and cleavage. Nat Commun 2021 12(1):3397.    

Liu Z, Kim HK, Xu J, Jing Y, Kay MA. The 3’tsRNAs are aminoacylated: implications for their biogenesis. PLoS Genet 2021 17(7):e1009675.    


Kim HK, Yeom JH, Kay MA. Transfer RNA-derived small RNAs: Another layer of gene regulation and novel targets for disease therapeutics. Mol Ther 2020 28(11):2341-2357.    

Su Y, Walker JR, Park Y, Smith TP, Liu LX, Hall MP, Labanieh L, Hurst R, Wang DC, Encell LP, Kim N, Zhang F, Kay MA, Casey KM, Majzner RG, Cochran JR, Mackall CL, Kirkland TA, Lin MZ. Novel NanoLuc substrates enable bright two-population bioluminescence imaging in animals. Nat Methods 2020 Aug;17(8):852-860.    

De Alencastro G, Pekrun K, Valdmanis P, Tiffany M, Xu J, Kay MA. Tracking Adeno-Associated Virus Capsid Evolution by High-Throughput Sequencing. Hum Gene Ther 2020 31(9-10):553-564.

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, Dupre N, Eichler EE, Dion PA, Rouleau GA, Valdmanis PN. Evolution of a human-specific tandem repeat associated with ALS. Am J Hum Genet 2020 107(3):445-460.  

Jackson CB, Richard AS, Ojha A, Conkright KA, Trimarchi JM, Bailey CC, Alpert MD, Kay MA, Farzan M, Choe H. AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery. Mol Ther Methods Clin Dev 2020 19:496-506.   


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. J Clin Invest Insight. 2019 14;4(22):e131610.   

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 Rep 2019 17;29(12):3816-3824.   


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. Nat Commun. 2018 9(1):5321.

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. Mol Ther Methods Clin Dev 2018 10:144-155.   

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. Mol Ther 2018 26(1):289-303.   


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 Comm 2017 8(1):2053.   

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.   

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. Sci Transl Med 2017 9(418).   

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. Mol Ther 2017 25(11):2477-2489.   

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 Mol Med 2017 9(10):1346-1355.   

Lu J, Zhang F, Fire AZ, Kay MA. Sequence-Modified Antibiotic Resistance Genes Provide Sustained Plasmid-Mediated Transgene Expression in Mammals. Mol Ther 2017 25(5):1187-1198.   

Valdmanis PN, Kay MA. Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond. Hum Gen Ther 2017 28(4):361-372.   

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. Nucl Acids Res 2017 45(1):311-326.   

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. Hum Gene Ther 2017 28(1):125-134.   


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. Exp Neurol 2016 286:137-146.   

Disterer P, Kay MA, Parker GC. Dieter C. Gruenert, PhD (1949-2016). Nucl Acids Ther 2016 26(4):266-267.   

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. Nat Biotechnol 2016 34(7):760-767.   

Nygaard S, Barzel A, Haft A, Major A, Finegold M, Kay MA, Grompe M. A universal system to select gene-modified hepatocytes in vivo. Sci Transl Med 2016 8(342):342ra79.   

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. Nat Med 2016 22(5):557-562.   

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. Hum Gen Ther 2016 27(4):280-286.   


Kay MA. Selecting the Best AAV Capsid for Human Studies. Mol Ther 2015 23(12):1800-1801.   

Mellins ED, Kay MA. Viral Vectors Take On HIV Infection. NEJM 2015 373(8):770-772.   

Haussecker D, Kay MA. RNA interference. Drugging RNAi. Science 2015 347(6226):1069-1070.   

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. Clin Dev 2015 26(1):5-14.   

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. Sci Rep 2015 5:8081.   

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):360-364.   

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.   


Nichols T, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA. Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs. Hum Gene Ther Clin Dev 2014 26:5-14   

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. Sci Transl Med 2014 6(264):264ra163.   

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. NEJM. 2014 371(21):1994-2004.   

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 Res 2014 42(19):12169-12176.   

Roy-Chaudhuri B, Valdmanis PN, Zhang Y, Wang Q, Luo QJ, Kay MA. Regulation of microRNA-mediated gene silencing by microRNA precursors. Nat Struct Mol Biol 2014 21(9):825-832.   

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. J Am Coll Cardiol 2014 64(5):451-459.   

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 Rep 2014 8(2):371-381.   

Phillips N, Kay MA. Characterization of vector-based delivery of neurogenin-3 in murine diabetes. Hum Gen Ther. 2014 25(7):651-61.   

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. Mol Ther 2014 22(5):952-963.   

Borel F, Kay MA, Mueller C. Recombinant AAV as a platform for translating the therapeutic potential of RNA interference. Mol Ther 2014 22(4):692-701.

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. Mol Ther 2014 22(4):725-33.   

Kay MA, Walker BD. Engineering cellular resistance to HIV. NEJM. 2014 370(10):968-969.   

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):382-386.   


Valdmanis PN, Kay MA. The expanding repertoire of circular RNAs. Mol Ther 2013 21(6):1112-1124.   

Lu J, Zhang F, Kay MA. A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Mol Ther 2013 21(5):954-963.   

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. Jpn J Clin 2013 43(5):579-582.   

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 Res 2013 41(6):3688-3698.

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. Mol Ther 2013 21(1):131-138.   


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. Circ Res 2012 111(12):1494-1503.   

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):900-911.   

Valdmanis PN, Lisowski L, Kay MA. rAAV-mediated tumorigenesis: still unresolved after an AAV assault. Mol Ther 2012 20(11):2014-2017.   

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. Mol Ther 2012 20(11):2111-2119.   

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. Mol Ther 2012 20(10):1912-23.   

Wang Z, Lisowski L, Finegold MJ, Nakai H, Kay MA, Grompe M. AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Mol Ther 2012 20(10):1902-1911.   

Gu S, Jin L, Huang Y, Zhang F, Kay MA. Slicing-independent RISC activation requires the argonaute PAZ domain. Curr Biol 2012 22(16):1536-1542.   

Valdmanis PN, Gu S, Schüermann N, Sethupathy P, Grimm D, Kay MA. Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucleic Acid Res 2012 40(8):3704-3713.   


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. NEJM 2011 365(25):2357-2365.   

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. J Clin Invest 2011 121(12):4850-60.   

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. Circ 2011 124(11 Suppl):S46-54.   

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. PNAS 2011 108(22):9208-13.   

Kay MA. State-of-the-art gene-based therapies: the road ahead. Nat Rev Genet 2011 12(5):316-328.   

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. Mol Ther 2011 19(3):450-60.   

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. Nat Protoc 2011 6(1):78-88.   

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 Transplant 2011 20(5):655-667.   


Kay MA, He CY, Chen ZY. A robust system for production of minicircle DNA vectors. Nat Biotechnol 2010 28(12):1287-9.   

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. Mol Ther 2010 18(11):1896-1906.   

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. J Clin Invest 2010 120(9):3106-3119.   

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. Endocrin Metab 2010 299(3):E384-93.   

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. Epigenet Chromatin 2010 3(1):13.   

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

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 2010 16 Suppl 3:19-23.   

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. Hepatol 2010 51(4):1200-1208.   

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 2010 16(4):673-695.

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. Nat Methods. 2010 7(3):197-199.   

Haussecker D, Kay MA. miR-122 continues to blaze the trail for microRNA therapeutics. Mol Ther. 2010 18(2):240-2.   

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. Mol Ther 2010 18(1):161-170.   


Cao D, Haussecker D, Huang Y, Kay MA. Combined proteomic-RNAi screen for host factors involved in human hepatitis delta virus replication. RNA 2009 15(11):1971-9.

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. Circ 2009 120(11 Suppl):S230-237.   

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. Nat Struct Mol Biol 2009 16(2):144-150.   

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. J Cardiovasc Pharmacol 2009 53(1):18-23.   

Jager L, Hausl MA, Rauschhuber C, Wolf NM, Kay MA, Ehrhardt A. A rapid protocol for construction and production of high-capacity adenoviral vectors. Nat Protoc 2009 4(4):547-564.   


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. Mol Ther 2008 16(9):1630-1636.   

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. J Biol Chem. 2008 283(32):22186-192.   

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. Hum Gene Ther 2008 19(8):820-6.   

Haussecker D, Cao D, Huang Y, Parameswaran P, Fire AZ, Kay MA. Capped small RNAs and MOV10 in human hepatitis delta virus replication. Nat Struct Mol Biol 2008 15(7):714-721.   

Rossi J, Zamore P, Kay MA. Wandering eye for RNAi. Nat Med 2008; 14(6):611.

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. J Virol 2008 82(12):5887-5911.   

Haussecker D. The business of RNAi therapeutics. Hum Gen Ther 2008 19(5):451-462.

Wilson JM, Gansbacher B, Berns KI, Bosch F, Kay MA, Naldini L, Wei YQ. Good news on the clinical gene transfer front. Hum Gen Ther 2008 19(5):429-430.   

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. Mol Ther 2008 16(5):931-941.   

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. Mol Ther 2008 16(3):548-556.   

Lazarus JJ, Kay MA, McCarter AL, Wooten RM. Viable Borrelia burgdorferi enhances interleukin-10 production and suppresses activation of murine macrophages. Infect Immun 2008 76(3):1153-1162.   

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 2008 47(2):706-718.   

Kay MA. Prime time for small RNA-based therapeutics. Hum Gen Ther 2008 19(1):15-16.   


Grimm D, Kay MA. Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? J Clin Invest 2007 117(12):3633-3641.   

Garrison BS, Yant SR, Mikkelsen JG, Kay MA. Postintegrative gene silencing within the Sleeping Beauty transposition system. Mol Cell Biol 2007 27(24):8824-8833.   

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. Hum Gen Ther 2007 18(12):1193-1204.   

Shen X, Storm T, Kay MA. Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency. Mol Ther 2007 15(11):1955-62.   

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. Hum Gen Ther 2007 18(10):871-880.   

Kay MA. AAV vectors and tumorigenicity. Nat Biotechnol 2007; 25(10):1111-1113.

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. J Virol 2007 81(20):11290-303.  

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. J Virol 2007 81(20):11304-11321.   

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. PNAS 2007 104(37):14771-6.   

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. Nat Biotechnol 2007 25(8):903-910.   

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. Mol Ther 2007 15(7):1273-1279.   

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. Mol Ther 2007 15(7):1348-1355.   

Grimm D, Kay MA. Combinatorial RNAi: a winning strategy for the race against evolving targets? Mol Ther 2007 15(5):878-888.   

Yant SR, Huang Y, Akache B, Kay MA. Site-directed transposon integration in human cells. Nucleic Acid Res 2007 35(7):e50.   

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 2007 25(2):371-379.   

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. Mol Ther 2007 15(2):255-263.   

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. Mol Ther 2007 15(2):330-339.   

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. Mol Ther 2007 15(1):146-156.   

Grimm D, Kay MA. RNAi and gene therapy: a mutual attraction. Hematology 2007 473-481.   


Ehrhardt A, Engler JA, Xu H, Cherry AM, Kay MA. Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration. Hum Gen Ther 2006 17(11):1077-1094.   

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. J Virol. 2006 80(19):9831-9836.   

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. Mol Ther 2006 14(1):45-53.   

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):537-541.   

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):4182-4188.   

Grimm D, Kay MA. Therapeutic short hairpin RNA expression in the liver: viral targets and vectors. Gen Ther 2006 13(6):563-575.  

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. Nat Med 2006 12(3):342-347.   

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. J Virol 2006 80(1):426-349.   


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. Transplant Proc 2005 37(10):4587-8.

Ehrhardt A, Kay MA. Gutted adenovirus: a rising star on the horizon? Gen Ther 2005 12(21):1540-1541.   

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. Mol Ther 2005 12(1):42-48.   

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. Mol Ther 2005 11(5):695-706.   

Riu E, Grimm D, Huang Z, Kay MA. Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo. Hum Gen Ther 2005 16(5):558-570.   

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. J Virol 2005 79(6):3606-3614.   

Yant SR, Wu X, Huang Y, Garrison B, Burgess SM, Kay MA. High-resolution genome-wide mapping of transposon integration in mammals. Mol Cell Biol 2005 25(6):2085-2094.   

Ohashi K, Nakai H, Couto LB, Kay MA. Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver. Hum Gen Ther 2005 16(3):299-306.   

Nakai H, Fuess S, Storm TA, Muramatsu S, Nara Y, Kay MA. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol 2005 79(1):214-224.   

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 2005 41(1):132-140.   

Grimm D, Pandey K, Kay MA. Adeno-associated virus vectors for short hairpin RNA expression. Meth Enzymol 2005 392:381-405.   

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. Hum Gen Ther 2005 16(1):126-131.   

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 Transplant 2005 14(9):621-627.   

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.   


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. J Vasc Interv Radiol 2004 15(11):1287-1293.   

Ohashi K, Kay MA. Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice. Transplant Proc 2004 36(8):2469-2470.   

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. Mol Cell Biol 2004 24(20):9239-47.   

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):530-536.   

Layzer JM, McCaffrey AP, Tanner AK, Huang Z, Kay MA, Sullenger BA. In vivo activity of nuclease-resistant siRNAs. RNA 2004 10(5):766-771.

Chen ZY, He CY, Meuse L, Kay MA. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gen Ther 2004 11(10):856-864.   

Wang AY, Peng PD, Ehrhardt A, Storm TA, Kay MA. Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo. Hum Gen Ther 2004 15(4):405-13.   

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. J Virol 2004 78(6):3110-3122.   


Yant SR, Kay MA. Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells. Mol Cell Biol 2003 23(23):8505-8518.   

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. Mol Ther 2003 8(4):654-665.   

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):2412-2419.   

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):2403-2411.   

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. Mol Ther 2003 8(3):495-500.   

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.   

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. Curr Gene Ther 2003 3(4):281-304.  

McCaffrey AP, Meuse L, Karimi M, Contag CH, Kay MA. A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice. Hepatology 2003 38(2):503-508.   

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. J Clin Invest 2003 112(3):407-414.   

Park F, Ohashi K, Kay MA. The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo. Mol Ther 2003 8(2):314-23.   

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

Nakai H, Montini E, Fuess S, Storm TA, Grompe M, Kay MA. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Gen 2003 34(3):297-302.   

Ehrhardt A, Xu H, Kay MA. Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo. J Virol 2003 77(13):7689-7695.   

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 Ther 2003 10(13):1099-104.   

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. Hum Gen Ther 2003 14(9):871-881.   

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. Mol Ther 2003 7(6):839-850.   

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. Nat Biotech 2003 21(6):639-644.   

Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Gen 2003 4(5):346-358.   

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):2963-2972.   

McCaffrey A, Kay MA, Contag CH. Advancing molecular therapies through in vivo bioluminescent imaging. Mol Imaging. 2003 2(2):75-86.   

Ehrhardt A, Peng PD, Xu H, Meuse L, Kay MA. Optimization of cis-acting elements for gene expression from nonviral vectors in vivo. Hum Gen Ther 2003 14(3):215-25.   

Sclimenti CR, Neviaser AS, Baba EJ, Meuse L, Kay MA, Calos MP. Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice. Biotechnol Prog 2003 19(1):144-151.   

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. Mol Ther 2003 7(1):101-111.  

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. Mol Ther 2003 7(1):112-121.   


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

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. Mol Ther 2002 6(6):759-69.   

Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP. Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol 2002 20(11):1124-1128.   

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. J Virol 2002 76(22):11343-11349.   

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. J Virol 2002 76(20):10465-10472.   

Yant SR, Ehrhardt A, Mikkelsen JG, Meuse L, Pham T, Kay MA. Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat Biotechnol 2002 20(10):999-1005.   

McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. RNA interference in adult mice. Nature 2002 418(6893):38-39.   

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):3923-3930.   

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. Mol Ther 2002;5(6):676-684.   

Ohashi K, Park F, Kay MA. Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo. Hum Gen Ther 2002 13(5):653-663.   


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. Mol Ther 2001 4(6):586-592.   

Ohashi K, Park F, Kay MA. Hepatocyte transplantation: clinical and experimental application. J Mol Med 2001 79(11):617-630.   

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. PNAS 2001 98(22):12584-12589.   

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. Mol Ther 2001 4(3):164-713.   

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. J Virol. 2001 75(15):6969-6976.   

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. Mol Ther 2001 4(2):122-129.   

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):1112-1114, 1116   

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. Gen Ther 2001 8(9):730-735.   

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. Mol Ther 2001 3(3):403-410.   

Ohashi K, Meuse L, Schwall R, Kay MA. cMet activation allows persistent engraftment of ectopically transplanted xenogenic human hepatocytes in mice. Transplant Proc 2001 33(1-2):587-588.   

Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med 2001 7(1):33-40.   


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. J Virol 2000 74(20):9451-9463.   

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):1173-1176.   

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. Mol Ther 2000 1(6):522-532.   

Nakai H, Storm TA, Kay MA. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat Biotechnol 2000 18(5):527-532.   

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. Nat Gen 2000 25(1):35-41.   

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. J Virol 2000 74(8):3793-3803.   

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. Nat Gen 2000 24(3):257-261.   

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. Nat Med 2000; 6(3):327-331.   

Park F, Ohashi K, Chiu W, Naldini L, Kay MA. Efficient lentiviral transduction of liver requires cell cycling in vivo. Nat Gen 2000 24(1):49-52.   

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. J Virol 2000 74(2):721-734.   


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. J Clin Endocrinol Metab 1999 84(12):4335-4340.   

Lieber A, Steinwaerder DS, Carlson CA, Kay MA. Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes. J Virol 1999 73(11):9314-24.   

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. J Biol Chem 1999 274(40):28808-15.   

Kay MA, High K. Gene therapy for the hemophilias. PNAS 1999; 96(18):9973-5.   

Mizuguchi H, Kay MA. A simple method for constructing E1- and E1/E4-deleted recombinant adenoviral vectors. Hum Gen Ther 1999 10(12):2013-7.   

Russell DW, Kay MA. Adeno-associated virus vectors and hematology. Blood. 1999; 94(3):864-874.   

Nakai H, Iwaki Y, Kay MA, Couto LB. Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. J Virol 1999 73(7):5438-5447.   

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. J Virol 1999 73(6):4755-4766.   

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

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. Nat Med 1999 5(1):64-70.   


Mizuguchi H, Kay MA. Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method. Hum Gen Ther 1998 9(17):2577-25783.   

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. J Virol 1998 72(11):9267-7927.   

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. J Virol 1998 72(9):7542-7550.   

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 1998 28(3):707-716.   

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

Kay MA. Hepatic gene therapy for haemophilia B. Haemophilia 1998 4(4):389-392.   

Patijn GA, Lieber A, Meuse L, Winther B, Kay MA. High-efficiency retrovirus-mediated gene transfer into the livers of mice. Hum Gen Ther 1998 9(10):1449-146.   

Miao CH, Snyder RO, Schowalter DB, Patijn GA, Donahue B, Winther B, Kay MA. The kinetics of rAAV integration in the liver. Nat Genet 1998 19(1):13-15.   


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. Am J Hum Genet 1997 61(6):1450-1454.   

Lieber A, He CY, Kay MA. Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo. Nat Biotechnol 1997 15(13):1383-1387.  

Kay MA, Liu D, Hoogerbrugge PM. Gene therapy. PNAS 1997 94(24):12744-12746.   

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. J Virol 1997 71(11):8798-8807.   

Nelson JE, Kay MA. Persistence of recombinant adenovirus in vivo is not dependent on vector DNA replication. J Virol 1997 71(11):8902-7.   

Schowalter DB, Kay MA. Gene therapy: a status report. Ped Ann 1997 26(9):562-568.   

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 Ther 1997 4(8):853-860.   

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. Nat Gen 1997 16(3):270-276.   

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

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. PNAS 1997 94(9):4686-4691.   

Vrancken Peeters MJ, Patijn GA, Lieber A, Perkins J, Kay MA. Expansion of donor hepatocytes after recombinant adenovirus-induced liver regeneration in mice. Hepatology 1997 25(4):884-888.   

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. Gen Ther 1997 4(4):351-360.   

Bethune C, Bui T, Liu ML, Kay MA, Ho RJ. Development of a high-performance liquid chromatographic assay for G418 sulfate (Geneticin). Antimicrob Agents CH 1997 41(3):661-664.   

Kay MA, Fausto N. Liver regeneration: prospects for therapy based on new technologies. Mol Med Today 1997 3(3):108-115.   

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 Ther 1997 4(1):9-16.   

Barr D, Kay MA. Methods for delivery of genes to hepatocytes in vivo using recombinant adenovirus vectors. Methods Mol Biol 1997 7:205-212.   


Thompson AR, Kay MA. Nonviral gene transfer to the liver. Hepatology 1996 24(6):1541-1542.   

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. J Virol 1996 70(12):8782-8791.   

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. J Virol 1996 70(12):8944-8960.   

Murry CE, Kay MA, Bartosek T, Hauschka SD, Schwartz SM. Muscle differentiation during repair of myocardial necrosis in rats via gene transfer with MyoD. J Clin Iinvest 1996 98(10):2209-2217.   

Sandig V, Löser P, Lieber A, Kay MA, Strauss M. HBV-derived promoters direct liver-specific expression of an adenovirally transduced LDL receptor gene. Gen Ther 1996 3(11):1002-1009.   

Peeters MJ, Patijn GA, Lieber A, Meuse L, Kay MA. Adenovirus-mediated hepatic gene transfer in mice: comparison of intravascular and biliary administration. Hum Gen Ther 1996 7(14):1693-1699.   

Lieber A, Kay MA. Adenovirus-mediated expression of ribozymes in mice. J Virol 1996 70(5):3153-3158.   

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. J Virol 1996 70(4):2497-502.   

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):278-285.   


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. Nat Genet1995 11(2):191-197.   

Wilson C, Kay MA. Immunomodulation to enhance gene therapy. Nat Med 1995 1(9):887-889.   

Lieber A, Peeters MJ, Gown A, Perkins J, Kay MA. A modified urokinase plasminogen activator induces liver regeneration without bleeding. Hum Gen Ther 1995 6(8):1029-1037.   

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. Hum Gen Ther 1995 6(8):1039-1044.   

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. PNAS 1995 92(13):6210-6214.   


Scaria A, Curiel DT, Kay MA. Complementation of a human adenovirus early region 4 deletion mutant in 293 cells using adenovirus-polylysine-DNA complexes. Gen Ther 1995 2(4):295-298.  

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. Gen Ther 1995 2(2):151-155.   

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 1995 21(3):815-819.   

Lieber A, Vrancken Peeters MJ, Kay MA. Adenovirus-mediated transfer of the amphotropic retrovirus receptor cDNA increases retroviral transduction in cultured cells. Hum Gen Ther 1995 6(1):5-11.   

Kay MA. Hepatic gene therapy for hemophilia B. Adv Exp Med Biol 1995 386:229-234.   


Kay MA, Woo SL. Gene therapy for metabolic disorders. Trends Genet 1994 10(7):253-257.   

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. PNAS 1994 91(6):2353-2357.   

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. PNAS 1993 90(24):11548-11552.   

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 1993 262(5130):117-119.   

Kay MA. Hepatocyte transplantation for liver gene therapy. Cell Transplant 1993 2(5):405-406.

Li Q, Kay MA, Finegold M, Stratford-Perricaudet LD, Woo SL. Assessment of recombinant adenoviral vectors for hepatic gene therapy. Hum Gen Ther 1993 4(4):403-409.   


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. Hum Gen Ther 1992 3(6):641-647.   

Liu TJ, Kay MA, Darlington GJ, Woo SL. Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice. Somat Cell Mol Genet 1992 18(1):89-96.   

Kay MA, Ponder KP, Woo SL. Human gene therapy: present and future. Breast Cancer Res Treat 1992 21(2):83-93.   

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. PNAS 1992 89(1):89-93.   


Kay MA, McCabe ED. Escherichia coli sepsis and prolonged hypophosphatemia following exertional heat stroke. Pediatrics 1990 86(2):307-309.   

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):589-592.   


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. Mol Gen Genet 1988 213(2-3):354-358.   


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 Res 1987 15(3):987-1003.  

Kay MA, Jacobs-Lorena M. Developmental genetics of ribosome synthesis in Drosophilai. Trends Genet 1987 3:347-351.   


Kay MA, Jacobs-Lorena M. Selective translational regulation of ribosomal protein gene expression during early development of Drosophila melanogaster. Mol Cell Biol 1985 5(12):3583-3592.