Kay Lab– in the department of Pediatrics & Genetics

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Publications

Peer-reviewed articles | Invited Manuscripts | Book Chapters

Titles link to downloadable PDF files.

Peer-reviewed articles

• Sebastiano, V., Zhen, H.H., Derafshi, B.H., Bashkirova, E., Melo, S.P., Wang, P., Leung, T.L., Siprashvili, Z., Tichy, A., Li, J., Ameen, M., Hawkins, J., Lee, S., Li, L., Schwertschkow, A., Bauer, G., Lisowski, L., M.A. Kay, Kim, S.K. Lane, A.T., Wernig, M., Oro, A.E . 2014. Human COL7A1-corrected induced pluriopotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa. Sci Transl Med 6(264):264.
• Nathwani, A.C., Reiss, U.M., Tuddenham, E.G., 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, Y.M., Halka, K.G., Basner-Tschakarajan, E., Mingozzi, F., High, K.A., Allay, J., M.A. Kay, Ng, C.Y., Zhou, J., Cancio, M., Morton, C.L., Gray, J.T., Srivastava, D., Nienhuis, A.W., Davidoff, A.M. 2014. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 371(21):1994-2004.
• Barzel, A., Paulk, N.K., Shi, Y., Huang, Y., Chu, K., Zhang, F., Valdmanis, P.N., Spector, L.P., Porteus, M.H., Gaensler, K.M., M.A. Kay. 2014. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Natur,e October 29 (Epub ahead of print)
• Gu, S., Zhang, Y., Jin, L., Huang, Y., Zhang, F., Bassik, M.C., Kampmann, M., M.A. Kay. 2014. Nucleic Acids Res 42(19):12169-12176.

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

• Wang, Y., Liang, P., Lan, F., Wu, H., Lisowski, L., Gu, M., Hu, S., M.A. Kay, Urnov, F.D., Shinnawi, R., Gold, J.D., Gepstein, L., Wu, J.C. 2014. Genome editing of isogenic human pluripotent stem cells recapitulates long QT phenotype for drug testing. J Am Coll Cardiol 64(5):451-459.

• Ehmer, U., Zmoos, A.F., Auerbach, R.K., Vaka, D., Butte, A.J., M.A. Kay, Sage, J. 2014. Organ size control is dominant over Rb family inactivation to restrict proliferation in vivo. Cell Rep 8(2):371-381.

• Phillips, N., M.A. Kay. 2014. Characterization of vector-based delivery of neurogenin-3 in murine diabetes. Hum Gene Ther 25(7):651-661.

• Kay, M.A., Walker, B.D. 2014. Engineering cellular resistance to HIV. N Engl J Med 370(10):968-969.

• Lisowski, L., Dane, A.P., Chu, K., Zhang, Y., Cunningham, S.C., Wilson, E.M., Nygaard, S., Grompe, M., Alexander, I.E., M.A. Kay. 2014. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 506(7488):382-386.

• Melo, S.P., Lisowski, L., Bashkirova, E., Zhen, H.H., Chu, K., Keene, D.R., Marinkovich, M.P., M.A. Kay, Oro, A.E. 2014. Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant. Mol Ther 22(4):725-733.

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

• Valdmanis, P.N., Roy-Chaudhuri, B., Kim, H.K., Sayles, L.C., Zheng, Y., Chuang, C.H., Caswell, D.R., Chu, K., Zhang, Y., Winslow, M.M., Sweet-Cordero, E.A., M.A. Kay. 2013. Upregulation of the microRNA cluster at the Dlk1-Dio3 locus in lung adenocarcinoma. Oncogene (Dec 9) Epub ahead of print.

• Lisowski, L., Elazar, M., Chu, K., Glenn, J.S., M.A. Kay. 2013. The anti-genomic (negative) strant of Hepatitis C Virus is not targetable by shRNA. Nucleic Acids Res 41(6):3688-3698.

• Valdmanis, P., M.A. Kay. 2013. The expanding repertoire of circular RNAs. Mol Ther 21(6):1112-1114.
• Lu, J., Zhang, F., M.A. Kay. 2013. A mini-intronic plasmid (MIP): A novel robust transgene expression vector in vivo and in vitro. Mol Ther 21(5):954-963.
• Wang, Z., Lisowski, L., Finegold, M.J., Nakai, H., M.A. Kay, Grompe, M. 2012. AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Mol Ther 20(10):1902-1911.
• Valdmanis, P.N., Lisowski, L., M.A. Kay. 2012. rAAV-mediated tumorigenesis: still unresolved after an AAV assault. Mol Ther 20(11):2014-2017.

• Lisowski, L., Lau, A., Wang, Z., Zhang, Y., Zhang, F., Grompe, M., M.A. Kay. 2012. Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Mol Ther 20(10):1912-1923.

• Wang, Y., Zhang, W.Y., Hu, S., Lan, F., Lee, A.S., Huber, B., Lisowski, L., Liang, P., Huang, M., de Almeida, P.E., Won, J.H., Sun, N., Robbins, R.C., M.A. Kay, UrnoV, F.D., Wu, J.C. 2012. Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging novelty and significance. Circ Res, 111:1494-150.

• Gu, S., Jin, L., Zhang, Y., Huang, Y., Zhang, F., Valdmanis, P.N., M.A .Kay. 2012. The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo. Cell, 151:900-911.
• Gracey Maniar, L., Maniar, J.M., Chen, Z-Y., Lu, J., Fire, A.Z., M.A. Kay. 2012. Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther, Nov 27 (ahead of pub).
• Valdmanis, P.N., Gu, S., Schurmann, N., Sethupathy, P., Grimm, D., Kay, M.A. 2012. Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucl Acids Res, 40(8):3704-3713.
• Lu, J., Zhang, F., Xu, S., Fire, A.Z., Kay, M.A. 2012. The Extragenic Spacer Length Between the 5′ and 3′ Ends of the Transgene Expression Cassette Affects Transgene Silencing From Plasmid-based Vectors. Mol Ther 20(11):2111-2119..
• Nathwani, A.C., Tuddenham, E.G.D., Rangarajan, S.,Rosales, C., McIntosh, J., Linch, D.C., Chir, B., Chowdary, P., Riddell, A., Pie, A.J., Harrington, C., O’Beirne, J., Smith, K., Pasi, J., Glader, B., Rustagi, P., Ng, C.Y.C., M.A. Kay, Zhou, J., Spence,Y., Morton, C.L ., Allay, J.,Coleman, J., Sleep,S., Cunningham, J.M., Srivastava, D., Basner-Tschakarjan,E., Mingozzi, F., High, K.A, Gray, J.T., Reiss, U.M., Nienhuis, A.W., Davidoff A.M. 2011. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B. N Engl J Med, 365:2357-2365.
• Osborn, M.J., R.T. McElmurry, C. J. Lees, A.P. DeFeo, Z.Y. Chen, M.A. Kay, L. Naldini, G. Freeman, J. Tolar, B.R. Blazar. 2011. Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of alpha-L-iduronidase in mice with mucopolysaccharidosis Type I. Mol Ther 19(3):450-460.
• Gu, S. L. Jin, F. Zhang, Y. Huang, D. Grimm, J.J. Rossi, M.A. Kay. 2011. Thermoodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes. Proc Nat Acad Sci 108(22):9208-9213.
• Kay, MA. 2011. State-of-the-art gene-based therapies: the road ahead. Nat Rev Gen 12(5):316-328.
• Narsinh, KH, F. Jia, R.C. Robbins, M.A. Kay, M.T. Longaker, J.C. Wu. 2011. Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Nat Prot 6(1):78-88.
• Kay, M.A., C.Y. He, Z.Y. Chen. 2010. A robust system for production of minicircle DNA vectors. Nat Biotechnol 28(12):1287-1289.
• Hausl, M.A., W. Zhang, N. Muther, C. Rauschhuber, H.G. Franck, E.P. Merricks, T.C. Nichols, M.A. Kay, A. Ehrhardt. 2010. Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B. Mol Ther 18(11):1896-1906.
• Grimm, D., L. Wang, J.S. Lee, N. Schurmann, S. Gu, K. Borner, T.A. Storm, M.A. Kay. 2010. Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest 120(9):3106-3119.
• Falcon, A., H. Doege, A. Fluitt, B. Tsang, N. Watson, M.A. Kay, A. Stahl. 2010. FATP2 is a hepatic fatty acid transporter and peroxisomal very long-chain acyl-CoA synthetase. 2010. Am J Physiol Endocrin Metab 299:E384-E393.
• Gracey, L.E., Z.Y. Chen, J.M. Maniar, A. Valouev, A. Sidow, M.A. Kay, A.Z. Fire. 2010. An in vitro-identified high-affinity nucleosome-positioning signal is capable of transiently positioning a nucleosome in vivo. Epigen Chrom 3(13):2-7.
• Nichols, T.C., R.A. Raymer, H.W.G. Franck, E.P. Merricks, D.A. Bellinger, N. Defriess, P. Margaritis, V.R., Arruda, M.A. Kay, K.A. High. 2010. Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B. Haemophilia 16(3):19-23.
• Gu, S. M.A. Kay. How do miRNAs mediate translational repression? 2010. Silence 1(1):11
• Paulk, N., K. Wursthorn , Z. Wang, M.J. Finegold, M.A. Kay, M. Grompe. 2010. Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology 51(4):1200-1208.
• Haussecker, D., Y. Huang, A. Lau, P. Parameswaran, A. Fire, M.A. Kay. 2010. Human tRNA-derived small RNAs in RNA silencing. RNA 16(4):673-695
• Jia, F., K.D. Wilson, N. Sun, D.M. Gupta, M. Huang, Z. Li, N.J. Panetta, R.C. Robbins, M.A. Kay, M.T. Longaker, J.C. Wu. 2010. A non-viral minicircle vector for deriving human iPS cells. Nature Methods 7(3):197-199
• Jager, L., M.A. Hausl, C. Rauschhuber, N.M. Wolf, M.A. Kay, A. Ehrhardt. 2009. A rapid protocol for construction and production of high-capacity adenoviral vectors. Nature Protocols 4:547-564.
• Cao, D., D. Haussecker, Y. Huang, M.A. Kay. 2009. Combined proteomic-RNAi screen for host factors involved in hepatitis delta virus replication. RNA 15:1971-1999.
• Huang, M., Z. Chen, S. Hu, F. Jia, G. Hoyt, R.C. Robbins, M.A. Kay, J.C. Wu. 2009. Novel minicircle vector gene therapy in murine myocardia infarction. Circulation 120(11 suppl): S230-237.
• Stenler, S., A. Andersson, O.E. Simonson, K.E. Lundin, Z.Y. Chen, M.A. Kay, C.I.E. Smith, C. Sylven, P. Blomberg. 2009. Gene Transfer to Mouse Heart and Skeletal Muscles Using a Minicircle Expressing Human Vascular Endothelial Growth Factor. J Cardiovasc Pharmacol, 53:18-23.
• Gu, S., L. Jin, F. Zhang, P. Sarnow, M.A. Kay. 2009. Biological basis for restriction of microRNA targets to the 3' untranslated region in mammalian mRNAs. Nat Struct Mol Biol, 16:144-1502.
• Giering, J.C., D. Grimm, T.A. Storm, M.A. Kay. 2008. Expression of shRNA from a Tissue-specific pol II Promoter Is an Effective and Safe RNAi Therapeutic. Mol Ther, 116:1630-6.
• Doege, H., D. Grimm, A. Falcon, B. Tsang, T.A. Storm, H. Xu, A.M. Ortegon, M. Kazantzis, M.A. Kay, A. Stahl. 2008. Silencing of Hepatic Fatty Acid Transporter Protein 5 in Vivo Reverses Diet-induced Non-alcoholic Fatty Liver Disease and Improves Hyperglycemia. J Biol Chem, 283:22186-92.
• McCaffrey, A.P., P. Fawcett, H. Nakai, R.L. McCaffrey, A. Ehrhardt, T.T. Pham, K. Pandey, H. Xu, S. Feuss, T.A. Storm, M.A. Kay. 2008. The Host Response to Adenovirus, Helper-dependent Adenovirus, and Adeno-associated Virus in Mouse Liver. Mol Ther, 16:931-41.

• Haussecker, D., D. Cao, Y. Huang, P. Parameswaran, A. Fire, M.A. Kay. 2008. Capped small RNAs and MOV10 in human hepatitis delta virus replication. Nat Struct Mol Biol, 15:714-21.
• Zhang, X., M.W. Epperly, M.A. Kay, Z.Y. Chen, T. Dixon, D. Franicola, P. Komanduri, B.A. Greenberger. 2008. Radioprotection In Vitro and In Vivo by Minicircle Plasmid Carrying the Human Manganese Superoxide Dismutase Transgene. Hum Gene Ther, 19:820-6.
• Grimm, D., J.S. Lee, L. Wang, T. Desai, B. Akache, T.A. Storm, M.A. Kay. 2008. In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Re-Targeting of Adeno-Associated Viruses. J Virol, June;82:5886-5911.
• Chen, Z.Y., E. Riu, C.Y. He, H. Xu, M.A. Kay. 2008. Silencing of Episomal Transgene Expression in Liver by Plasmid Bacterial Backbone DNA Is Independent of CpG Methylation. Mol Ther, 16:548-56.
• Streetz, K.L., R. Doyonnas, D. Grimm, D.D. Jenkins, S. Fuess, S. Perryman, J. Lin, C. Trautwein, J. Shizuru, H. Blau, K.G. Sylvester, M.A. Kay. 2008. Hepatic Parenchymal Replacement in Mice by Transplanted Allogenic Hepatocytes Is Facilitated by Bone Marrow Transplantation and Mediated by CD4 Cells. Hepatology, 47:706-18.
• Azuma H., N. Paulk, A. Ranade, C. Dorrell, M. Al-Dhalimy, E. Ellis, S. Strom, M.A. Kay, M. Finegold, M. Grompe. 2007. Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/112rg-/- mice. Nat Biotechnol, 25:903-10.
• Moldt B., S.R. Yant, P.R. Anderson, M.A. Kay, J.G Mikkelson. 2007, Cis-Acting Gene Regulatory Activities in the Terminal Regions of Sleeping Beauty DNA Transposon-Based Vectors. Hum Gene Ther, 18:1193-204.
• Grimm, D., M.A. Kay. 2007, Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? J Clin Invest, 117:3633-41.
• Garrison, B.S., S.R. Yant, J.G. Mikkelsen, M.A. Kay. 2007, Postintegrative Gene Silencing within the Sleeping Beauty Transposition System. Mol Cell Biol, 27:8824-33.
• Paskowitz, D.M., K.P. Greenberg, D. Yasumura, D. Grimm, H. Yang, J.L. Duncan, M.A. Kay, M.M. Lavail, J.G. Flannery, D. Vollrath. 2007, Rapid and Stable Knockdown of an Endogenous Gene in Retinal Pigment epithelium. Hum Gene Ther, 18:871-80.
• Tward, A.D., K.D. Jones, S. Yant, S.T. Cheung, S.T. Fan, X. Chen, M.A. Kay, R. Wang, J.M. Bishop. 2007, Distinct pathways of genomic progression to benign and malignant tumors of the liver. Proc Natl Acad Sci USA, 104:14771-14776.
• Shen, X., T.A. Storm, M.A. Kay. 2007, Characterization of the Relationship of AAV Capsid Domain Swapping to Liver Transduction Efficiency. Mol Ther, 15:1955-62.
• Inagaki, K., C. Ma, T.A. Storm, M.A. Kay, H. Nakai. 2007, The Role of DNA-PKcs and Artemis in Opening Viral DNA Hairpin Termini in Various Tissues in Mice. J Virol, 81:11304-21.
• Inagaki, K., S.M. Lewis, X. Wu, C. Ma, D.J. Munroe, S. Fuess, T.A. Storm, M.A. Kay, H. Nakai. 2007, DNA Palindromes with a Modest Arm Length of >=~20 Base Pairs Are a Significant Target for rAAV Vector Integration in the Liver, Muscle, and Heart in Mice. J Virol, 81:11290-303.
• Azuma, H., N. Paulk, A. Ranade, C. Dorrell, M. Al-Dhalimy, E. Ellis, S. Storm, M.A. Kay, M. Finegold, M. Grompe . 2007, Robust expansion of human hepatocytes in Fah(-/-)/Rag2(-/-)/Il2rg(-/-) mice. Nat Biotechnol, 25:903-10.
• Riu, E., Z.Y. Chen, H. Xu, C.Y. He, M.A. Kay. 2007, Histone Modifications Are Associated with the Persistence or Silencing of Vector-Mediated Transgene Expression In Vivo. Mol Ther, 15:1348-55.
• Zayed, H., L. Xia, A. Yerich, S.R. Yant, M.A. Kay, M. Puttaraju, G.J. McGarrity, D.L. Wiest, R.S. McIvor, J. Tolar, B.R. Blazar. 2007, Correction of DNA Protein Kinase Deficiency by Spliceosome-Mediated RNA Trans-splicing and Sleeping Beauty Transposon Delivery. Mol Ther, 15:1273-79.
• Yant, S.R., Y. Huang, B. Akache, M.A. Kay. 2007, Site-Directed Transposon Integration in Human Cells. Nucleic Acids Res, 35:e50.
• Grimm, D., M.A. Kay. 2007, Combinatorial RNAi: A Winning Strategy for the Race against Evolving Targets? Mol Ther, 15:878-88.
• Akache, B., D. Grimm, X. Shen, S. Fuess, S.R. Yant, D.S. Glazer, J. Park, M.A. Kay. 2007, A Two-Hybrid Screen Identifies Cathepsins B and L as Uncoating Factors for Adeno-associated Virus 2 and 8. Mol Ther, 15:330-9.
• Wang, A.Y., A. Ehrhardt, H. Xu, M.A. Kay. 2007, Adenovirus Transduction Is Required for the Correction of Diabetes Using Pdx-1 or Neurogenin-3 in the Liver. Mol Ther, 15:255-63.
• Ehrhardt, A., S.R. Yant, J.C. Giering, H. Xu, J.A. Engler, M.A. Kay. 2007, Somatic Integration from an Adenoviral Hybrid Vector into a Hot Spot in Mouse Liver Results in Persistent Transgene Expression Levels In Vivo. Mol Ther, 15:146-56.
• Ehrhardt, A., J.A. Engler, H. Xu, A.M. Cherry, M.A. Kay. 2006, Molecular Analysis of Chromosomal Rearrangements in Mammalian Cells after phiC31-Mediated Integration. Hum Gene Ther, 17:1077-94.
• Tolar, J., A.J. Nauta, M.J. Osborn, A. Panoskaltsis Mortari, R.T. McElmurry, S. Bell, L. Xia, N. Zhou, M. Riddle, T.M. Schroeder, J.J. Westendorf, R.S. McIvor, P.C. W. Hogendoorn, K. Szuhai, L. Oseth, B. Hirsch, S.R. Yant, M.A. Kay, A. Peister, D.J. Prockop, W.E. Fibbe, B.R. Blazar. 2007, Sarcoma Derived from Cultured Mesenchymal Stem Cells. Stem Cells, 25:371-379.
• Akache, B., D. Grimm, K. Pandey, S.R. Yant, H. Xu, M.A. Kay. 2006, The 37/67-Kilodalton Laminin Receptor Is a Receptor for Adeno-Associated Virus Serotypes 8, 2, 3, and 9. J Virol, 80:9831-6.
• Inagaki, K., S. Fuess, T.A. Storm, G.A. Gibson, C.F. Mctiernan, M.A. Kay, H. Nakai. 2006, Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer Superior to that of AAV8. Mol Ther, 14:45-53.
• Grimm, D., M.A. Kay. 2006, Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype. J Virol, 80:426-39.
• Tolar, J., M.J. O'Shaughnessy, A. Panoskaltsis-Mortari, R.T. McElmurry, S. Bell, M. Riddle, R.S. McIvor, S.R. Yant, M.A. Kay, D. Krause, C.M. Verfaillie, B.R. Blazar. 2006, Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells. Blood, 107:4182-8.
• Grimm, D., K.L. Streetz, C.L. Jopling, T.A. Storm, K. Pandey, C.R. Davis, P. Marion, F. Salazar, M.A. Kay. 2006, Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature, 441:537-541.
• Manno, C.S., G.F. Pierce, V.R. Arruda, B. Glader, M. Ragni, J. Rasko, M.C. Ozelo, K. Hoots, P. Blatt, B. Konkle, M. Dake, R. Kaye, M. Razavi, A. Zajko, J. Zehnder, P.K. Rustagi, H. Nakai, A. Chew, D. Leonard, J.F. Wright, R.R. Lessard, J.M. Sommer, M. Tigges, D. Sabatino, A. Luk, H. Jiang, F. Mingozzi, L. Couto, H.C. Ertl, K.A. High, M.A. Kay. 2006, Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med, 12:342-7.
• Grimm, D., K. Pandey, H. Nakai, T.A. Storm, M.A. Kay. 2006, Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype. J Virol, 80:426-39.
• Tolar, J., M. Osborn, S. Bell, R. McElmurry, L. Xia, M. Riddle, A. Panoskaltsis-Mortari, Y. Jiang, R.S. McIvor, C.H. Contag, S.R. Yant, M.A. Kay, C.M. Verfaillie, B.R. Blazar. 2005. Real-Time in Vivo Imaging of Stem Cells Following Transgenesis by Transposition. Mol Ther, 12:42-8.
• Riu, E., D. Grimm, Z. Huang, M.A. Kay. 2005. Increased Maintenance and Persistence of Transgenes by Excision of Expression Cassettes from Plasmid Sequences In Vivo. Hum Gene Ther, 16:558-570.
• Ohashi, K., M.A. Kay, T. Yokoyama, H. Kuge, H. Kanehiro, M. Hisanaga, S. Ko, Y. Nakajima. 2005. Stability and Repeat Regeneration Potential of the Engineered Liver Tissues Under the Kidney Capsule in Mice. Cell Transplant, 14:621-7.
• Ohashi, K., M.A. Kay, H. Kuge, T. Yokoyama, H. Kanehiro, M. Hisanaga, S. Ko, M. Nagao, M. Sho, Y. Nakajima. 2005. Heterotopically Transplanted Hepatocyte Survival Depends on Extracellular Matrix Components. Transplant Proc, 37:4587-8.
• Ohashi, K., H. Nakai, L.B. Couto, M.A. Kay. 2005. Modified Infusion Procedures Affect Recombinant Adeno-Associated Virus Vector Type 2 Transduction in the Liver. Hum Gene Ther, 16:299-306.
• Ehrhardt, A., H. Xu, Z. Huang, M.A. Kay. 2005. 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, 11:695-706.
• Yant, S.R., X. Wu, Y. Huang, B. Garrison, S.M. Burgess, M.A. Kay. 2005. High-Resolution Genome-Wide Mapping of Transposon Integration in Mammals. Mol Cell Biol, 25:2085-2094.
• Chen Z.Y., C.Y. He, M.A. Kay. 2005. Improved Production and Purification of Minicircle DNA Vector Free of Plasmid Bacterial Sequences and Capable of Persistent Transgene Expression In Vivo. Hum Gene Ther, 16:126-131
• Nakai, H., X. Wu, S. Fuess, T.A. Storm, D. Munroe, E. Montini, S.M. Burgess, M. Grompe, M.A. Kay. 2005. Large-Scale Molecular Characterization of Adeno-Associated Virus Vector Integration in Mouse Liver. J Virol, 79:3606-3614.
• Nakai, H., S. Fuess, T.A. Storm, S. Muramatsu, Y. Nara, M.A. Kay. 2005. Unrestricted Hepatocyte Transduction with AAV Serotype 8 Vectors in Mice. J Virol, 79:214–224.
• Ohashi, K., J.M. Waugh, M.D. Dake, T. Yokoyama, H. Kuge, Y. Nakajima, M. Yamanouchi, H. Naka, A. Yoshioka, M.A. Kay. 2005. Liver Tissue Engineering at Extrahepatic Sites in Mice as a Potential New Therapy for Genetic Liver Diseases. Hepatology, 41:132-140.
• Yant, S.R., J. Park, Y. Huang, J.G. Mikkelsen, M.A. Kay. 2004. 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, 24:9239-9247.
• Jenkins, D.D., K. Streetz, M. Tataria, D. Sahar, M. Kurobe, M.T. Longaker, M.A. Kay, K.G. Sylvester. 2004. Donor-Derived, Liver-Specific Protein Expression after Bone Marrow Transplantation. Transplantation, 78:530-536.
• Layzer, J.M., A.P. McCaffrey, A.K. Tanner, Z. Huang, M.A. Kay, B.A. Sullenger. 2004. In vivo activity of nuclease-resistant siRNAs. RNA, 10:766-771.
• Wang A.Y., P. Peng, A. Ehrhardt, T.A. Storm, M.A. Kay. 2004, Comparison of Adenoviral and Adeno-Associated Viral Vectors for Pancreatic Gene Delivery In VivoHum Gene Ther, 15:405-413.
• Thomas, C.E., T.A. Storm, Z. Huang, M.A. Kay. 2003, Rapid Uncoating of Vector Genomes Is the Key to Efficient Liver Transduction with Pseudotyped Adeno-Associated Virus Vectors. J Virol, 78:3110-3122.
• Chen, Z.Y., C.Y. He, Meuse L., M.A. Kay. 2003. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Ther, 11:856-864.
• Yant, S.R., M.A. Kay. 2003. Non-Homologous End-Joining Factors Regulate DNA Repair Fidelity during Sleeping Beauty Element Transposition in Mammalian Cells. Mol Cell Biol, 23:8505-8518.
• Bordier B., J. Ohkanda, P. Liu, S.Y. Lee, F.H. Salazar, P.L. Marion, K. Ohashi, L. Meuse, M.A. Kay, J.L. Casey, S.M. Sebti, A.D. Hamilton, J.S. Glenn. 2003. In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus. J Clin Invest, 112:407-414.
• Mikkelsen, J. G., S.R. Yant, L. Meuse, Z. Huang, H. Xu, M.A. Kay. 2003. Helper-Independent Sleeping Beauty Transposon-Transposase Vectors for Efficient Nonviral Gene Delivery and Persistent Gene Expression in Vivo. Mol Ther, 8:654-665.
• Chyung Y.H., P. Peng, M.A. Kay. 2003. System for Simultaneous Tissue-Specific and Disease-Specific Regulation of Therapeutic Gene Expression. Hum Gene Ther, 14:1255-1264.
• McCaffrey A.P., L. Meuse, M. Karimi, C.H. Contag, M.A. Kay. 2003, A Potent and Specific Morpholino Antisense Inhibitor of Hepatitis C Translation in Mice. Hepatology, 38:503-508.
• Ehrhardt, A., H. Xu, A.M. Dillow, D.A. Bellinger, T.C. Nichols, M.A. Kay. 2003, A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood, 102:2403-2411.
• Grimm D., S. Zhou, H. Nakai, C.E. Thomas, T.A. Storm, S. Fuess, T. Matsushita, J. Allen, R. Surosky, M. Lochrie, L. Meuse, A. McClelland, P. Colosi, M.A. Kay. 2003, Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood, 102:2412-19.
• Park, F., K. Ohashi, M.A. Kay. 2003, The Effect of Age on Hepatic Gene Transfer with Self-Inactivating Lentiviral Vectors in Vivo. Mol Ther, 8:314-23.
• Nakai, H., T.A. Storm, S. Fuess, M.A. Kay. 2003, Pathways of Removal of Free DNA Vector Ends in Normal and DNA-PKcs-Deficient SCID Mouse Hepatocytes Transduced with rAAV Vectors. Hum Gene Ther, 14:871-881
• Nakai, H., E. Montini, S. Fuess, T.A. Storm, M. Grompe, M.A. Kay. 2003, AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet, 34:297-304.
• Grimm, D., M.A. Kay, J.A. Kleinschmidt. 2003, Helper Virus-Free, Optically Controllable, and Two-Plasmid-Based Production of Adeno-associated Virus Vectors of Serotypes 1 to 6. Mol Ther, 7:839-850.

• Chen, Z.Y., C.Y. He, A. Ehrhardt, M.A. Kay. 2003, Minicircle DNA Vectors Devoid of Bacterial DNA Result in Persistent and High-Level Transgene Expression in Vivo. Mol Ther, 8:495-500.
• Ehrhardt, A., H. Xu, M.A. Kay. 2003, Episomal Persistence of Recombinant Adenoviral Vector Genomes during the Cell Cycle in Vivo. J Virol, 77:7689-7695.
• McCaffrey, A.P., H. Nakai, K. Pandey, Z. Huang, F.H. Salazar, H. Xu, P.L. Marion, M.A. Kay. 2003, Inhibition of hepatitis B virus replication in mice by RNA interference. Nat Biotechnol, 21:639-644.
• Ortiz-Urda S., Q. Lin, S.R. Yant, D. Keene, M.A. Kay, P.A. Khavari. 2003, Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer. Gene Ther, 10:1099-1104.
• Manno, C.S., A.J. Chew, S. Hutchinson, P.J. Larsen, R. Herzog, V.R. Arruda, S.J. Tai, M.V. Ragni, A. Thompson, M. Ozelo, L.B. Couto, D.G.B. Leonard, F. Johnson, A. McClelland, C. Scallan, E. Skarsgard, A.W. Flake, M.A. Kay, K.A. High, B. Glader. 2003, AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood, 101: 2963-2972.
• Ehrhardt A., P. Peng, H. Xu, L. Meuse, M.A. Kay. 2003, Optimization of CIS-Acting Elements for Gene Expression from Nonviral Vectors in Vivo. Hum Gene Ther, 14:215-225.
• Nakai, H., S. Fuess, T.A. Storm, L.A. Meuse, M.A. Kay. 2003, Free DNA Ends Are Essential for Concatemerization of Synthetic Double-Stranded Adeno-Associated Virus Vector Genomes Transfected into Mouse Hepatocytes. Mol Ther, 1:112-121.
• Nakai, H. Montini, S. Feuss, T.A. Storm, L. Meuse, M. Finegold, M. Grompe, M.A Kay. 2003, Helper-Independent and AAV-ITR-Independent Chromosomal Integration of Double-Stranded DNA Vectors in Mice. Mol Ther, 1:101-111.
• Montini E., P., K. Held, M. Noll, N. Morcinek, M. Al-Dhalimy, M. Finegold, S. R. Yant, M.A. Kay, M. Grompe. 2002, In Vivo Correction of Murine Tyrosinemia Type I by DNA-Mediated Transposition. Mol Ther, 6:759-769.
• Olivares, E.C., R.P. Hollis, T.W. Chalberg, L. Meuse, M.A. Kay, M.P. Calos. 2002, Site-specific integration produces therapeutic Factor IX levels in mice. Nat Biotechnol, 20:1124-1128.
• Yant, S., A. Ehrhardt, J.G. Mickelson, L. Meuse, T. Pham, M.A. Kay. 2002, Transposition from a gutless adenovirus-transposon vector stabilizes transgene expression in vivo. Nat Biotechnol, 20:999-1005.
• Nakai, H., C.E. Thomas, T.A. Storm, S. Feuss, S. Powell, J.F. Wright, M.A. Kay. 2002, A Limited Number of Transducible Hepatocytes Restricts a Wide-Range Linear Vector Dose Response in rAAV-Mediated Liver Transduction. J Virol, 76:11343-11349.
• McCaffrey, A.P., L. Meuse, T.T. Pham, D.S. Conklin, G.J. Hannon, M.A. Kay. 2002, Gene Expression: RNA interference in adult mice. Nature, 418:38-39.
• McCaffrey, A.P., K. Ohashi, L. Meuse, S. Shen, A.M. Lancaster, P.J. Lukavsky, M.A. Kay. 2002, Determinants of Hepatitis C Translational Initiation in Vitro, in Cultured Cells and Mice. Mol Ther, 5:676-684.
• Ohashi, K., F. Park, R.H. Schwall, M.A. Kay. 2002, Efficient Gene Transduction to Cultured Hepatocytes by HIV-1 Derived Lentiviral Vector. Transplant Proc, 24:1431-1433.
• Bordier, B.B., P.L. Marion, K. Ohashi, M.A. Kay, H.B. Greenberg, J.L. Casey, J.S. Glenn. 2002, A Prenylation Inhibitor Prevents Production of Infectious HDV Particles. J Virol, 76:10465-10472.
• Ehrhardt, A., M. A. Kay. 2002, A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood, 99: 3923-3930.
• Ohashi, K., F. Park, M. A. Kay. 2002, Role of Hepatocyte Direct Hyperplasia on Lentivirus-Mediated Liver Transduction in Vivo. Hum Gene Ther, 13:653-663.
• Arruda, V.R., P.A. Fields, R. Milner, L. Wainwright, M.P. De Miguel, P.J. Donovan, R.W. Herzog, T.C. Nichols, J.A. Biegel, M. Razavi, M. Dake, D. Huff, A.W. Flake, L. Couto, M.A. Kay, K.A. High. 2001, Lack of Germline Transmission of Vector Sequences Following Systemic Administration of Recombinant AAV-2 Vector in Males. Mol Ther, 4:586-592.
• Vollrath, D., W. Feng, J.L. Duncan, D. Yasumura, P.M. D'Cruz, A. Chappelow, M.T. Matthes, M.A. Kay, M.M. La Vail. 2001, Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc Natl Acad Sci USA, 98:12584-12589.
• Nakai, H, S.R. Yant, T.A. Storm, S. Fuess, L. Meuse, M.A. Kay. 2001, Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo. J Virol, 75:6969-76.
• Park, F., M.A. Kay. 2001, Modified HIV-1 Based Lentiviral Vectors Have an Effect on Viral Transduction and Gene Expression in Vitro and in Vivo. Mol Ther, 4:164-73.
• Chen, Z.Y., S.R. Yant, C.Y. He, L. Meuse, S. Shen, M.A. Kay. 2001, Linear DNAs Concatemerize in Vivo and Result in Sustained Transgene Expression in Mouse Liver. Mol Ther, 3:403-410.
• Stoll, S.M., C.R. Sclimenti, E.J. Baba, L. Meuse, M.A. Kay, M.P. Calos. 2001, Epstein-Barr Virus/Human Vector Provides High-Level, Long-Term Expression of Alpha 1-Antitrypsin in Mice. Mol Ther, 4:122-129.
• Mizuguchi, H., M.A. Kay, T. Hayakawa. 2001, In Vitro Ligation-Based Cloning of Foreign DNAs into the E3 and E1 Deletion Regions for Generation of Recombinant Adenovirus Vectors. Biotechniques, 30:1112-1116.
• Mizuguchi, H., N. Koizumi, T. Hosono, N. Utoguchi, Y. Watanabe, M.A. Kay, T. Hayakawa. 2001, A simplified system for constructing recombinant adenoviral vectors containing heterologous peptides in the HI Loop of their fiber knob. Gene Ther, 8:730-735.
• Ohashi, K., L. Meuse, R. Schwall, M.A. Kay. 2001, cMet Activation Allows Persistent Engraftment of Ectopically Transplanted Xenogenic Human Hepatocytes in Mice. Transplant Proc, 33:587-588.
• Nakai, H., T. Storm, and M. A. Kay. 2000, Recruitment of Single-Stranded Recombinant Adeno-Associated Virus Vector Genomes and Intermolecular Recombinantion Are Responsible for Stable Transduction of Liver in Vivo. J Virol, 74:9451-9463.
• Miao, C.H., K. Ohashi, G.A. Patijn, L. Meuse, X. Ye, A.R. Thompson, M.A. Kay. 2000, Inclusion of the Hepatic Locus Control Region, an Intron, and Untranslated Region Enhances Hepatic Factor IX Gene Expression in Vivo but not in Vitro. Mol Ther, 1:432-442.
• Nakai, H., T.A. Storm, M.A. Kay. 2000, Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat Biotechnol, 18:527-532.
• Yant, S.R., L. Meuse, W. Chiu, Z. Ivics, Z. Izsvak, M.A. Kay. 2000, Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat Genet, 25:35-41.
• Park, F., K. Ohashi, M.A. Kay. 2000, Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood, 96:1173-1176.
• Miao, C.H., H. Nakai, A.R. Thompson, T.A. Storm, W. Chiu, R.O. Snyder, M.A. Kay. 2000, Nonrandom Transduction of Recombinant Adeno-Associated Viral Vectors in Mouse Hepatocytes in Vivo: Cell Cycling Is Not Required for Transduction. J Virol, 74:3793-3803.
• Kay, M.A., C.S. Manno, M.V. Ragni, P.J. Larson, L.B. Couto, A. McCleland, B. Glader, A.J. Chew, S.J. Tai, R.W. Herzog, V. Arruda, F. Johnson, C. Scallan, E. Skarsgard, A.W. Flake, K.A. High. 2000, Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet, 24:257-261.
• Ohashi, K., P.L. Marion, H. Nakai, L. Meuse, J.M. Cullen, B.B. Bordier, R. Schwall, H.B. Greenberg, J.S. Glenn, and M.A. Kay. 2000, Sustained survival of human hepatocytes: A model for in vivo infection with human hepatitis B and hepatitis delta viruses. Nat Med, 6:327-331.
• Park, F., K. Ohashi, W. Chiu, L. Nandini, M.A. Kay. 2000, Efficient lentiviral transduction of liver requires cell cycling in vivo. Nat Genet, 24:49-52.
• Lieber, A., M.A. Kay, Z.Y. Li. 2000, Nuclear Import of Moloney Murine Leukemia Virus DNA Mediated by Adenoviral Preterminal Protein Is Not Sufficient for Efficient Retroviral Transduction in Nondividing Cells. J Virol, 74:721-734.
• Vilain, E., M. Le Merrer, C. Lecointre, F. Desangles, M.A. Kay, P. Maroteaux, E.R.B. McCabe. 1999, IMAGe, a New Clinical Association of Intrauterine Growth Retardation, Metaphyseal Dysplasia, Adrenal Hypoplasia Congenita and GEnital Anomalies. J Clin Endocrinol Metab, 84:4335-4340.
• Lieber, A., D.S. Steinwaerder, C.A. Carlson, M.A. Kay. 1999, Integrating Adenovirus-Adeno-Associated Virus Hybrid Vectors Devoid of All Genes. J Virol, 73:9314-9324.
• Zen, K., A. Karsan, A. Stempien-Otero, E. Yee, J. Tupper, X. Li, T. Eunson, M. A. Kay, C. B. Wilson, R.K. Winn, J.M. Harlan. 1999, NF-Kappa-B Activation Is Required for Human Endothelial Survival during Exposure to Tumor Necrosis Factor-Alpha but Not to Interleukin-1-Beta or Lipopolysaccharide. J Biol Chem, 274:28808-28815.
• Mizuguchi, H., M. A. Kay. 1999, A Simple Method for Constructing E1- and E1/E4-Deleted Recombinant Adenoviral Vectors. Hum Gene Ther, 10:2013-2017.
• Schowalter, D.D., C.L. Himeda, B.L. Winther, C.B. Wilson, M.A. Kay. 1999, 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, 73:4755-4766.
• Nakai, H., Y. Iwaki, M.A. Kay, L.B. Couto. 1999, Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver. J Virol, 73:5438-5447.
• Snyder, R., C. Miao, L. Meuse, J. Tubb, B.A. Donahue, H.F. Lin, D.W. Stafford, S. Patel, A.R. Thompson, T. Nichols, M. Read, D.A. Bellinger, K.M. Brinkhous, M.A. Kay. 1999, Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med, 5:64-70.
• Stempien-Otero, A., A. Karsan, C.J. Cornejo, H. Xiang, T. Eunson, R. Morrison, M.A. Kay, C.B. Wilson, R. Winn, J. Harlan. 1999, Mechanisms of Hypoxia-Induced Endothelial Cell Death: Role of p53 in Apoptosis. J Biol Chem, 274:8039-8045.
• Mizuguchi, H., M.A. Kay. 1998, Efficient Construction of a Recombinant Adenovirus Vector by an Improved In Vitro Ligation Method. Hum Gene Ther, 9:2577-2583.
• Lieber, A., C.Y. He, L. Meuse, C. Himeda, C. Wilson, M.A. Kay. 1998, 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, 72:9267-9277.
• Miao, C. H., R. Snyder, D.B. Schowalter, G.A. Patijn, B. Donahue, B. Winther, M.A. Kay. 1998, The kinetics of rAAV integration in the liver. Nat Genet, 19:13-15.
• Wilson, C.B., L.J. Embree, D. Schowalter, R. Albert, A. Aruffo, D. Hollenbaugh, P. Linsley, M.A. Kay. 1998, 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, 72:7542-7550.
• Patijn, G., O. Terpstra, M.A. Kay. 1998, Method for continous perfusion into the livers of mice. Lab Anim Sci, 48:379-383.
• Patijn, G.A., A. Lieber, L. Meuse, B. Winther, M.A. Kay. 1998, High-Efficiency Retrovirus-Mediated Gene Transfer into the Livers of Mice. Hum Gene Ther, 9:1449-1456.
• Patijn, G.A., A. Lieber, D.B. Schowalter, R. Schwall, M.A. Kay. 1998, Hepatocyte Growth Factor (HGF) Induces Hepatocyte Proliferation In Vivo and Allows for Efficient Retroviral-Mediated Gene Transfer in Mice. Hepatology, 28:707-716.
• Lieber, A., C.Y. He, M.A. Kay. 1997, Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo. Nat Biotechnol, 15:1383-1387.
• Lieber, A., C.Y. He, L. Meuse, D. Schowalter, I. Kirillova, B. Winther, M.A. Kay. 1997, The Role of Kupffer Cell Activation and Viral Gene Expression in Early Liver Toxicity following Infusion of Recombinant Adenoviral Vectors. J Virol, 71:8798-8807.
• Nelson, J. E., M.A. Kay. 1997, Persistence of Recombinant Adenovirus In Vivo Is Not Dependent on Vector DNA Replication. J Virol, 71:8902-8907.
• Bennett, R.L., M. Karayiorgou, C.A. Sobin, T.H. Norwood, M.A. Kay. 1997, 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, 61:1450-1454.
• Schowalter, D.B., L. Meuse, C.B. Wilson, P.S. Linsley, M.A. Kay. 1997, Constitutive expression of murine CTLA4Ig from a recombinant adenovirus vector results in prolonged transgene expression. Gene Ther, 4:853-860
• Snyder, R.O., C.H. Miao, G.J. Patijn, S.K. Pratt, O. Danos, A.M. Gown, B. Winther, L. Meuse, L.K. Cohen, A.R. Thompson, M.A. Kay. 1997, Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet, 16:270-276
• Kay, M.A., L. Meuse, A.M. Gown, P. Linsley, D. Hollenbaugh, A. Aruffo, H. Ochs, C.B. Wilson. 1997, Transient immunomodulation with anti-CD40 ligand and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver. Proc Natl Acad Sci USA, 94:4686-4691
• Overturf, K., M. Al-Dhalimy, C.N. Ou, M. Finegold, R. Tanguay, A. Lieber, M.A. Kay, M. Grompe. 1997, Adenovirus-Mediated Gene Therapy in a Mouse Model of Hereditary Tyrosinemia Type 1. Hum Gene Ther, 8:513-521.
• Bethune, C., T. Bui, M L. Liu, M.A. Kay, R.J.Y. Ho. 1997, Development of a High-Performance Liquid Chromatographic Assay for G418 Sulfate (Geneticin). Antimicrob Agents Chemother, 41:661-4.
• Vrancken Peeters, M.J., G.A. Patijn, A. Lieber, J. Perkins, M.A. Kay. 1997, Expansion of Donor Hepatocytes after Recombinant Adenovirus-Induced Liver Regeneration in Mice. Hepatology, 25:884-888
• Schowalter, D.B., J.C. Tubb, M. Liu, C.B. Wilson, M.A. Kay. 1997, 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 Ther, 4:351-60.
• Kay, M.A., N. Fausto. 1997, Liver regeneration: prospects for therapy based on new technologies. Mol Med Today, 3:108-115.
• Brand, K., W. Arnold, T. Bartels, A. Lieber, M.A. Kay, M. Strauss, and B. Dorken. 1997, Toxicity associated with the treatment of liver tumors using the HSV-tk/GCV system and adenoviruses as vectors. Cancer Gene Ther, 4:9-16
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• Lieber, A., C.Y. He, I. Kirillova, M.A. Kay. 1996, 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, 70:8944-8960.
• Murry, C.E., M.A. Kay,T. Bartosek, S.D. Hauschka, S.M. Schwartz. 1996, Muscle Differentiation during Repair of Myocardial Necrosis via Gene Transfer with MyoD. J Clin Invest, 98:2209-2217.
• Vrancken Peeters, M.J., G. Patijin, A. Lieber, L. Meuse, M.A. Kay. 1996, Adenovirus-Mediated Hepatic Gene Transfer in Mice: Comparison of Intravascular and Biliary Administration. Hum Gene Ther, 7:1693-1699.
• Sandig, V., P. Liser, A. Lieber, M.A. Kay, M. Strauss. 1996, HBV-Derived Promoters Direct Liver-Specific Expression of an Adenovirus-Transduced LDL Receptor Gene. Gene Ther, 3:1002-1009.
• Lieber, A., C.Y. He, S. Polyack, D. Gretch, D. Barr, M.A. Kay. 1996, Elimination of Hepatitis C Virus RNA in Infected Human Hepatocytes by Adenovirus-Mediated Expression of Ribozymes. J Virol, 70:8782-8791.
• Grim, J., J. Deshane, M. Feng, A. Lieber, M.A. Kay, D. T. Curiel. 1996, ErbB-2 knockout employing an intracellular single chain antibody (sFv) accomplishes specific toxicity in erbB-2 expressing lung cancer cells. Am J Respir Cell Mol Biol, 15:348-354.
• Lieber, A. M.A. Kay. 1996, Adenovirus-Mediated Expression of Ribozymes in Mice. J Virol, 70:3153-3158.
• Liu, M.L., B. Winther, M.A. Kay. 1996, 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, 70:2497-2502.
• Vrancken-Peeters, M.J., A. Lieber, J. Perkins, M.A. Kay. 1996, Method for Multiple Portal Vein Infusions in Mice: Quantitation of Adenovirus-Mediated Hepatic Gene Transfer. BioTechniques, 20:278-285.
• Deshane, J., G.P. Siegal, R.D. Alvarez, M. Wang, M. Feng, G. Cabrera, T. Liu, M.A. Kay, D.T. Curiel. 1995, Targeted Tumor Killing via an Intracellular Antibody Directed against erbB-2. J Clin Invest, 96:2980-2989.
• Kay, M.A., A.X. Holterman, L. Meuse, A. Gown, H. Ochs, P.S. Linsley, C.B. Wilson. 1995, Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration. Nat Genet, 11:191-197.
• Fang, B., R.C. Eisensmith, H. Wang, M.A. Kay, G. Zhao, R.E. Cross, C.N. Landen, G. Gavin, D.A. Bellinger, M.S. Read, P.C. Hu, K.M. Brinkhous, S.L.C. Woo. 1995, Gene Therapy for Hemophilia B: Host immunosuppression Prolongs the Therapeutic Effect of Adenovirus-Mediated Factor IX Expression. Hum Gene Ther, 6:1039-1044
• Lieber, A., M.J. Vrancken Peeters, A. Gown, J. Perkins, M.A. Kay. 1995, A Modified Urokinase Plasminogen Activator Induces Liver Regeneration without Bleeding. Hum Gene Ther, 6:1029-1037.
• Lieber, A., M.J. Vrancken Peeters, L. Meuse, N. Fausto, J. Perkins, M.A. Kay. 1995, Adenovirus-mediated urokinase gene transfer induces liver regeneration and allows for efficient retrovirus transduction of hepatocytes in vivo. Proc Natl Acad Sci USA, 92:6210-6214
• Scaria, A., D.T. Curiel, M.A. Kay. 1995, Complementation of a human adenovirus early region 4 deletion mutant in 293 cells using adenovirus-polylysine-DNA complexes. Gene Ther, 2:295-298
• Lieber, A., M.J. Vrancken Peeters, M.A. Kay. 1995, Adenovirus-Mediated Transfer of the Rat Retrovirus Amphotropic Receptor cDNA Increases Retrovirus Transduction in Cultured Cells. Hum Gene Ther, 6:5-11
• Barr, D., J. Tubb, D. Ferguson, A. Scaria, A. Lieber, C. Wilson, J. Perkins, M.A. Kay. 1995, Strain related variations in adenoviral-mediated transgene expression from mouse hepatocytes in vivo: Comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther, 2:151-156.
• Kay, M.A., F. Graham, F. Leland, S.L.C. Woo. 1995, Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology, 21:515-519.
• Jones, D., M.A. Kay, W. Craigen, E. McCabe, H. Hawkins, A. Dominey. 1995, Coal-black hyperpigmentation at birth in a child with congenital adrenal hypoplasia. J Am Acad Dermatol, 33:323-326.
• Kay, M.A., C.N. Lander, S.R. Rothenberg, L.A. Taylor, F. Leland, S. Wiehle, B. Fang, D. Bellenger, M. Finegold, A.R. Thompson, M.S. Read, K.M. Brinkhous, S.L.C. Woo. 1994, In vivo hepatic gene therapy: Complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc Natl Acad Sci USA, 91:2353-2357.
• Cristiano, R.J., L.C. Smith, M.A. Kay, B.R. Brinkley, S.L.C. Woo. 1993, Hepatic gene therapy: Efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex. Proc Natl Acad Sci USA, 90:11548-11552.
• Kay, M.A., S. Rothenberg, C. Landon, D. Bellinger, F. Leland, C. Toman, A. Thompson, M. Read, K. Brinkhous, S.L.C. Woo. 1993, In Vivo Gene Therapy of Hemophilia B: Sustained partial correction in Factor IX-Deficient Dogs. Science, 262:117-119.
• Kolodka, T.M., M. Finegold, M.A. Kay, S.L.C. Woo. 1993, Hepatic Gene Therapy: Efficient Retroviral-Mediated Gene Transfer into Rat Hepatocytes In Vivo. Somat Cell Mol Genet, 19:491-497. Erratum appears in Vol 20:3 (1994).
• Li, Q.T., M A. Kay, L.S. Perricaudet, M. Finegold, S.L.C. Woo. 1993, Assessment of Recombinant Adenoviral Vectors for Hepatic Gene Therapy. Hum Gene Ther, 4:403-409.
• Kay, M.A., Q.T. Li, T.J. Liu, F. Leland, M. Finegold, S.L.C. Woo. 1992, Hepatic Gene Delivery: Persistent Expression of Human Alpha-1-Antitrypsin in Mice after Direct Gene Delivery In Vivo. Hum Gene Ther, 3:641-647.
• Liu, T.J., M.A. Kay, G.J. Darlington, S.L.C. Woo. 1992, Reconstitution of Enzymatic Activity in Hepatocytes of Phenylalanine Hydroxylase-Deficient Mice. Somat Cell Mol Genet, 18:89-96.
• Kay, M.A., P. Baley, S. Rothenberg, F. Leland, L. Fleming, K. Ponder, T.J. Liu, M. Finegold, G. Darlington, W. Pokorny, S.L.C. Woo. 1992, Expression of human alpha-1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA, 89:89-93.
• Kay, M.A., E.R.B. McCabe. 1990, Escherichia Coli Sepsis and Prolonged Hypophosphatemia Following Exertional Heat Stroke. Pediatrics, 86:307-309.
• Kay, M.A., W. O'Brien, B. Kessler, R. McVie, S. Ursin, K. Dietrich, E.R.B. McCabe. 1990, Transient organic aciduria and methemoglobinemia with acute gastroenteritis. Pediatrics, 85:589-592.
• Kay, M.A., J.Y. Zhang, M. Jacobs-Lorena. 1988, Identification and germ line transformation of the ribosomal protein rp21 gene of Drosophila: Complementation analysis with the Minute QIII locus reveals nonidentity. Mol Gen Genet, 213:354-358.
• Kay, M.A., and M. Jacobs-Lorena. 1987, Developmental genetics of ribosome synthesis in Drosophila. Trends Genet, 3:347-351.
• Qian, S., J.Y. Zhang, M.A. Kay, M. Jacobs-Lorena. 1987, Structure analysis of the Drosophila rpA1 gene, a member of the eucaryotic 'A' type ribosomal protein family. Nucleic Acids Res, 15:987-1003.
• Kay, M.A., M. Jacobs-Lorena. 1985, Selective translational regulation of ribosomal protein gene expression during early development of Drosophila Melanogaster. Mol Cell Biol, 5:3583-3592.

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Invited Manuscripts

• Kay, M.A., K.P. Ponder, S.L.C. Woo. 1992, Human gene therapy: present and future. Breast Cancer Research and Treatment, 21:83-93.
• Kay, M. A. 1993, Hepatocyte transplantation for liver gene therapy. Cell Transplantation, 2:405-406.
• Kay, M.A., S.L.C. Woo. 1994, Gene therapy for metabolic disorders. Trends Genet, 10:253-257.
• Wilson, C., M.A. Kay. 1995, Immunomodulation to enhance gene therapy. News and Views. Nat Med, 1:887-889.
• Kay, M.A. 1995, Hepatic gene therapy for hemophilia B. Adv Exp Med Biol, 386:229-334.
• Thompson, A., M.A. Kay. 1996, Nonviral Gene Transfer to the Liver. Hepatology, 24:1541-1542.
• Kay, M.A. 1997, Adenoviral Vectors for Hepatic Gene Transfer in Animals. Chest, 111:138S-142S.
• Schowalter, D.B., M.A. Kay. 1997, Gene Therapy: A Status Report. Pediatric Annals, 26:562-568.
• Kay, M.A., D. Liu, P.M. Hoogerbrugge. 1997, Gene Therapy. Proc Natl Acad Sci USA, 94:12744-12746.
• Russell, D.W., M.A. Kay. 1999, Adeno-Associated Virus Vectors and Hematology. Blood, 94:864-874.
• Patijn, G.A., M.A. Kay. 1999, Hepatic Gene Therapy using Adeno-Associated Virus (AAV) Vectors. Seminars in Liver Disease, 19:61-69.
• Kay, M.A., K. High. 1999, Gene therapy for the hemophilias. Proc Natl Acad Sci, 96:9973-9975.
• Kay, M.A., J.C. Glorioso, L. Naldini. 2001, Viral Vectors: The art of turning infectious agents into vehicles of therapeutics. Nat Med, 7:33-40.
• Mizuguchi, H., M.A. Kay, and T. Hayakawa. 2001, Approaches for generating recombinant adenoviral vectors. Adv Drug Deliv Rev, 52:165-176.
• Ohashi, K., F. Park, M.A. Kay. 2002, Hepatocyte transplantation: clinical and experimental Application. J Mol Med, 79:617-30.
• McCaffrey, A.P., M.A. Kay. 2002, A story of mice and men. Gene Ther, 9:1563.
• Thomas C.E., A. Ehrhardt, M.A. Kay. 2003, Progress and Problems with the Use of Viral Vectors for Gene Therapy. Nat Rev Genet, 4:346-358.
• Grimm, D., M. A. Kay. 2003, 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, 3:281-305.
• McCaffrey A.P., M.A. Kay, C.H. Contag, 2003, Advancing Molecular Therapies through In Vivo Bioluminescent Imaging. Mol Imaging, 2:75-86.
• McCaffrey A.P., M.A. Kay. 2003, RNA interference gets infectious. Gene Ther, 10:1205.
• Nakai, H., M.A. Kay. 2003, Looking into the safety of AAV vectors. Nature, 424:251.
• Ohashi, K., M.A. Kay. 2004, Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice. Transplant Proc, 36:2469-70.
• Grimm, D., K. Pandey, M.A. Kay. 2005, Adeno-Associated Virus Vectors for Short Hairpin RNA Expression. Methods Enzymol, 392:381-405.
• Ehrhardt, A., M.A. Kay. 2005, Gutted adenovirus: a rising star on the horizon? Gene Ther, 12:1540-1541.
• Grimm, D., M.A. Kay. 2006, Therapeutic short hairpin RNA expression in the liver: viral targets and vectors. Gene Ther, 13:563-75.
• M.A. Kay. 2007, AAV vectors and tumorigenicity. Nat Biotechnol, 25:1111-3.

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Book Chapters

• Barr, D. and M.A. Kay. Methods for delivery of genes to hepatocytes in vivo using recombinant adenovirus vectors. in Gene Therapy Protocols. ed. Paul Robbins Methods in Molecular Medicine 1997 Humana Press: Totowa, N.J.
• Kay, M.A. Hepatic Gene Therapy for Hemophilia B. in Inhibitors to Coagulation Factors. Advances in Experimental Medicine and Biology vol. 386. L.M.Aledort, L.W. Hoyer, J.M. Lusher, H.M. Reisner and G.C. White II.Plenum Press: NY pp 229-234.
• Kay, M.A. and D. Russell Chapter 69 Gene Therapeutics for Harrison's Textbook of Medicine 2001 edition.
• Kay, M.A., Chapter 71, Gene Therapy. Nelson Textbook of Pediatrics, (2003) 17th edition. R.E. Behrman, R. M. Kliegman, H.B. Jenson. Suanders: An imprint of Elsevier Science: PA pp 391-395.
• Grimm D., K. Pandey, M.A. Kay., Adeno-associated virus vectors for short hairpin RNA expression. A Chapter in RNA Interference, (Volume 392 of Methods in Enzymology), edited by J. Rossi and D.R. Engelke: An imprint of Elsevier Science. Chapter 23.

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