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Mark Kay
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30
Meuse, Leonard
22
Storm, Theresa
20
Yant, Stephen
19
Nakai, Hiroyuki
16
Grimm, Dirk
15
Lieber, André
13
Fuess, Sally
13
Xu, Hui
12
Ehrhardt, Anja
12
Ohashi, Kazuo
10
He, Cheng-Yi
9
Finegold, Milton
9
Patijn, GA
7
Thompson, Arthur
7
Woo, SL
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All Publications
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2009: Huang Mei; Chen ZhiYing; Hu Shijun; Jia Fangjun; Li Zongjin; Hoyt Grant; Robbins Robert C; Kay Mark A; Wu Joseph C
Novel minicircle vector for gene therapy in murine myocardial infarction.
Circulation 2009;120(11 Suppl):S230-7.
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2008: Giering Jeffery C; Grimm Dirk; Storm Theresa A; Kay Mark A
Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(9):1630-6.
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2008: Zhang Xichen; Epperly Michael W; Kay Mark A; Chen Zhi-Ying; Dixon Tracy; Franicola Darcy; Greenberger Benjamin A; Komanduri Paavani; Greenberger Joel S
Radioprotection in vitro and in vivo by minicircle plasmid carrying the human manganese superoxide dismutase transgene.
Human gene therapy 2008;19(8):820-6.
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2008: Doege Holger; Grimm Dirk; Falcon Alaric; Tsang Bernice; Storm Theresa A; Xu Hui; Ortegon Angelica M; Kazantzis Melissa; Kay Mark A; Stahl Andreas
Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia.
The Journal of biological chemistry 2008;283(32):22186-92.
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2008: Haussecker Dirk; Cao Dan; Huang Yong; Parameswaran Poornima; Fire Andrew Z; Kay Mark A
Capped small RNAs and MOV10 in human hepatitis delta virus replication.
Nature structural & molecular biology 2008;15(7):714-21.
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2008: Grimm Dirk; Lee Joyce S; Wang Lora; Desai Tushar; Akache Bassel; Storm Theresa A; Kay Mark A
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses.
Journal of virology 2008;82(12):5887-911.
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2008: McCaffrey Anton P; Fawcett Paul; Nakai Hiroyuki; McCaffrey Ramona L; Ehrhardt Anja; Pham Thu-Thao T; Pandey Kusum; Xu Hui; Feuss Sally; Storm Theresa A; Kay Mark A
The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(5):931-41.
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2008: Chen Zhi-Ying; Riu Efren; He Chen-Yi; Xu Hui; Kay Mark A
Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(3):548-56.
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2008: Streetz Konrad L; Doyonnas Regis; Grimm Dirk; Jenkins D Denison; Fuess Sally; Perryman Scott; Lin Joseph; Trautwein Christian; Shizuru Judith; Blau Helen; Sylvester Karl G; Kay Mark A
Hepatic parenchymal replacement in mice by transplanted allogeneic hepatocytes is facilitated by bone marrow transplantation and mediated by CD4 cells.
Hepatology (Baltimore, Md.) 2008;47(2):706-18.
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2007: Grimm Dirk; Kay Mark A
Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?
The Journal of clinical investigation 2007;117(12):3633-41.
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2007: Moldt Brian; Yant Stephen R; Andersen Peter Refsing; Kay Mark A; Mikkelsen Jacob Giehm
Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors.
Human gene therapy 2007;18(12):1193-204.
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2007: Garrison Brian S; Yant Stephen R; Mikkelsen Jacob Giehm; Kay Mark A
Postintegrative gene silencing within the Sleeping Beauty transposition system.
Molecular and cellular biology 2007;27(24):8824-33.
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2007: Shen Xuan; Storm Terry; Kay Mark A
Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(11):1955-62.
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2007: Kay Mark A
AAV vectors and tumorigenicity.
Nature biotechnology 2007;25(10):1111-3.
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2007: Paskowitz Daniel M; Greenberg Kenneth P; Yasumura Douglas; Grimm Dirk; Yang Haidong; Duncan Jacque L; Kay Mark A; Lavail Matthew M; Flannery John G; Vollrath Douglas
Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium.
Human gene therapy 2007;18(10):871-80.
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2007: Inagaki Katsuya; Ma Congrong; Storm Theresa A; Kay Mark A; Nakai Hiroyuki
The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice.
Journal of virology 2007;81(20):11304-21.
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2007: Inagaki Katsuya; Lewis Susanna M; Wu Xiaolin; Ma Congrong; Munroe David J; Fuess Sally; Storm Theresa A; Kay Mark A; Nakai Hiroyuki
DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice.
Journal of virology 2007;81(20):11290-303.
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2007: Tward Aaron D; Jones Kirk D; Yant Stephen; Cheung Siu Tim; Fan Sheung Tat; Chen Xin; Kay Mark A; Wang Rong; Bishop J Michael
Distinct pathways of genomic progression to benign and malignant tumors of the liver.
Proceedings of the National Academy of Sciences of the United States of America 2007;104(37):14771-6.
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2007: Azuma Hisaya; Paulk Nicole; Ranade Aarati; Dorrell Craig; Al-Dhalimy Muhsen; Ellis Ewa; Strom Stephen; Kay Mark A; Finegold Milton; Grompe Markus
Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice.
Nature biotechnology 2007;25(8):903-10.
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2007: Riu Efren; Chen Zhi-Ying; Xu Hui; He Chen-Yi; Kay Mark A
Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(7):1348-55.
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2007: Zayed Hatem; Xia Lily; Yerich Anton; Yant Stephen R; Kay Mark A; Puttaraju M; McGarrity Gerard J; Wiest David L; McIvor R Scott; Tolar Jakub; Blazar Bruce R
Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(7):1273-9.
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2007: Grimm Dirk; Kay Mark A
Combinatorial RNAi: a winning strategy for the race against evolving targets?
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(5):878-88.
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2007: Akache Bassel; Grimm Dirk; Shen Xuan; Fuess Sally; Yant Stephen R; Glazer Dariya S; Park Julie; Kay Mark A
A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(2):330-9.
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2007: Wang Alfred Y; Ehrhardt Anja; Xu Hui; Kay Mark A
Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(2):255-63.
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2007: Tolar Jakub; Nauta Alma J; Osborn Mark J; Panoskaltsis Mortari Angela; McElmurry Ron T; Bell Scott; Xia Lily; Zhou Ning; Riddle Megan; Schroeder Tania M; Westendorf Jennifer J; McIvor R Scott; Hogendoorn Pancras C W; Szuhai Karoly; Oseth Leann; Hirsch Betsy; Yant Stephen R; Kay Mark A; Peister Alexandra; Prockop Darwin J; Fibbe Willem E; Blazar Bruce R
Sarcoma derived from cultured mesenchymal stem cells.
Stem cells (Dayton, Ohio) 2007;25(2):371-9.
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2007: Ehrhardt Anja; Yant Stephen R; Giering Jeffery C; Xu Hui; Engler Jeffrey A; Kay Mark A
Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(1):146-56.
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2007: Yant Stephen R; Huang Yong; Akache Bassel; Kay Mark A
Site-directed transposon integration in human cells.
Nucleic acids research 2007;35(7):e50.
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2006: Ehrhardt Anja; Engler Jeffrey A; Xu Hui; Cherry Athena M; Kay Mark A
Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration.
Human gene therapy 2006;17(11):1077-94.
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2006: Akache Bassel; Grimm Dirk; Pandey Kusum; Yant Stephen R; Xu Hui; Kay Mark A
The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9.
Journal of virology 2006;80(19):9831-6.
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2006: Inagaki Katsuya; Fuess Sally; Storm Theresa A; Gibson Gregory A; Mctiernan Charles F; Kay Mark A; Nakai Hiroyuki
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8.
Molecular therapy : the journal of the American Society of Gene Therapy 2006;14(1):45-53.
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2006: Grimm Dirk; Streetz Konrad L; Jopling Catherine L; Storm Theresa A; Pandey Kusum; Davis Corrine R; Marion Patricia; Salazar Felix; Kay Mark A
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.
Nature 2006;441(7092):537-41.
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2006: Tolar Jakub; O'shaughnessy Matthew J; Panoskaltsis-Mortari Angela; McElmurry Ron T; Bell Scott; Riddle Megan; McIvor R Scott; Yant Stephen R; Kay Mark A; Krause Diane; Verfaillie Catherine M; Blazar Bruce R
Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells.
Blood 2006;107(10):4182-8.
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2006: Grimm D; Kay M A
Therapeutic short hairpin RNA expression in the liver: viral targets and vectors.
Gene therapy 2006;13(6):563-75.
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2006: Grimm Dirk; Pandey Kusum; Nakai Hiroyuki; Storm Theresa A; Kay Mark A
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype.
Journal of virology 2006;80(1):426-39.
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2005: Tolar Jakub; Osborn Mark; Bell Scott; McElmurry Ron; Xia Lily; Riddle Megan; Panoskaltsis-Mortari Angela; Jiang Yuehua; McIvor R Scott; Contag Christopher H; Yant Stephen R; Kay Mark A; Verfaillie Catherine M; Blazar Bruce R
Real-time in vivo imaging of stem cells following transgenesis by transposition.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;12(1):42-8.
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2005: Riu Efren; Grimm Dirk; Huang Zan; Kay Mark A
Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo.
Human gene therapy 2005;16(5):558-70.
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2005: Ehrhardt Anja; Xu Hui; Huang Zan; Engler Jeffrey A; Kay Mark A
A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;11(5):695-706.
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2005: Ohashi Kazuo; Nakai Hiroyuki; Couto Linda B; Kay Mark A
Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver.
Human gene therapy 2005;16(3):299-306.
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2005: Yant Stephen R; Wu Xiaolin; Huang Yong; Garrison Brian; Burgess Shawn M; Kay Mark A
High-resolution genome-wide mapping of transposon integration in mammals.
Molecular and cellular biology 2005;25(6):2085-94.
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2005: Nakai Hiroyuki; Wu Xiaolin; Fuess Sally; Storm Theresa A; Munroe David; Montini Eugenio; Burgess Shawn M; Grompe Markus; Kay Mark A
Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver.
Journal of virology 2005;79(6):3606-14.
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2005: Chen Zhi-Ying; He Cheng-Yi; Kay Mark A
Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo.
Human gene therapy 2005;16(1):126-31.
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2005: Ohashi Kazuo; Waugh Jacob M; Dake Michael D; Yokoyama Takashi; Kuge Hiroyuki; Nakajima Yoshiyuki; Yamanouchi Masaki; Naka Hiroyuki; Yoshioka Akira; Kay Mark A
Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases.
Hepatology (Baltimore, Md.) 2005;41(1):132-40.
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2005: Nakai Hiroyuki; Fuess Sally; Storm Theresa A; Muramatsu Shin-ichi; Nara Yuko; Kay Mark A
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice.
Journal of virology 2005;79(1):214-24.
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2005: Tward A D; Jones K D; Yant S; Kay M A; Wang R; Bishop J M
Genomic progression in mouse models for liver tumors.
Cold Spring Harbor symposia on quantitative biology 2005;70():217-24.
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2005: Grimm Dirk; Pandey Kusum; Kay Mark A
Adeno-associated virus vectors for short hairpin RNA expression.
Methods in enzymology 2005;392():381-405.
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2004: Ganaha Fumikiyo; Ohashi Kazuo; Do Young So; Lee Jane; Sugimoto Koji; Minamiguchi Hiroki; Elkins Christopher J; Sameni Diba; Modanlou Shoreh; Ali Mir; Kao Edward Y; Kay Mark A; Waugh Jacob M; Dake Michael D
Efficient inhibition of in-stent restenosis by controlled stent-based inhibition of elastase: a pilot study.
Journal of vascular and interventional radiology : JVIR 2004;15(11):1287-93.
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2004: Ohashi K; Kay M A
Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice.
Transplantation proceedings 2004;36(8):2469-70.
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2004: Yant Stephen R; Park Julie; Huang Yong; Mikkelsen Jacob Giehm; Kay Mark A
Mutational analysis of the N-terminal DNA-binding domain of sleeping beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells.
Molecular and cellular biology 2004;24(20):9239-47.
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2004: Jenkins D Denison; Streetz Konrad; Tataria Monika; Sahar David; Kurobe Masashi; Longaker Michael T; Kay Mark A; Sylvester Karl G
Donor-derived, liver-specific protein expression after bone marrow transplantation.
Transplantation 2004;78(4):530-6.
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2004: Chen Z Y; He C Y; Meuse L; Kay M A
Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo.
Gene therapy 2004;11(10):856-64.
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2004: Wang Alfred Y; Peng Peter D; Ehrhardt Anja; Storm Theresa A; Kay Mark A
Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo.
Human gene therapy 2004;15(4):405-13.
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2004: Thomas Clare E; Storm Theresa A; Huang Zan; Kay Mark A
Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors.
Journal of virology 2004;78(6):3110-22.
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2003: Yant Stephen R; Kay Mark A
Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells.
Molecular and cellular biology 2003;23(23):8505-18.
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2003: Mikkelsen Jacob Giehm; Yant Stephen R; Meuse Leonard; Huang Zan; Xu Hui; Kay Mark A
Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;8(4):654-65.
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2003: Ehrhardt Anja; Xu Hui; Dillow Aaron M; Bellinger Dwight A; Nichols Timothy C; Kay Mark A
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia.
Blood 2003;102(7):2403-11.
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2003: Grimm Dirk; Zhou Shangzhen; Nakai Hiroyuki; Thomas Clare E; Storm Theresa A; Fuess Sally; Matsushita Takashi; Allen James; Surosky Richard; Lochrie Michael; Meuse Leonard; McClelland Alan; Colosi Peter; Kay Mark A
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy.
Blood 2003;102(7):2412-9.
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2003: Chyung Yung H; Peng Peter D; Kay Mark A
System for simultaneous tissue-specific and disease-specific regulation of therapeutic gene expression.
Human gene therapy 2003;14(13):1255-64.
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2003: Chen Zhi-Ying; He Cheng-Yi; Ehrhardt Anja; Kay Mark A
Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;8(3):495-500.
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2003: Bordier Bruno B; Ohkanda Junko; Liu Ping; Lee So-Young; Salazar F H; Marion Patricia L; Ohashi Kazuo; Meuse Leonard; Kay Mark A; Casey John L; Sebti Said M; Hamilton Andrew D; Glenn Jeffrey S
In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus.
The Journal of clinical investigation 2003;112(3):407-14.
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2003: McCaffrey Anton P; Meuse Leonard; Karimi Mobin; Contag Christopher H; Kay Mark A
A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice.
Hepatology (Baltimore, Md.) 2003;38(2):503-8.
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2003: Grimm D; Kay M A
From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy.
Current gene therapy 2003;3(4):281-304.
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2003: Ortiz-Urda S; Lin Q; Yant S R; Keene D; Kay M A; Khavari P A
Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer.
Gene therapy 2003;10(13):1099-104.
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2003: Ehrhardt Anja; Xu Hui; Kay Mark A
Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo.
Journal of virology 2003;77(13):7689-95.
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2003: Nakai Hiroyuki; Montini Eugenio; Fuess Sally; Storm Theresa A; Grompe Markus; Kay Mark A
AAV serotype 2 vectors preferentially integrate into active genes in mice.
Nature genetics 2003;34(3):297-302.
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2003: Nakai Hiroyuki; Storm Theresa A; Fuess Sally; Kay Mark A
Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors.
Human gene therapy 2003;14(9):871-81.
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2003: Grimm Dirk; Kay Mark A; Kleinschmidt Juergen A
Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;7(6):839-50.
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2003: McCaffrey Anton P; Nakai Hiroyuki; Pandey Kusum; Huang Zan; Salazar Felix H; Xu Hui; Wieland Stefan F; Marion Patricia L; Kay Mark A
Inhibition of hepatitis B virus in mice by RNA interference.
Nature biotechnology 2003;21(6):639-44.
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2003: Thomas Clare E; Ehrhardt Anja; Kay Mark A
Progress and problems with the use of viral vectors for gene therapy.
Nature reviews. Genetics 2003;4(5):346-58.
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2003: McCaffrey Anton; Kay Mark A; Contag Christopher H
Advancing molecular therapies through in vivo bioluminescent imaging.
Molecular imaging : official journal of the Society for Molecular Imaging 2003;2(2):75-86.
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2003: Manno Catherine S; Chew Amy J; Hutchison Sylvia; Larson Peter J; Herzog Roland W; Arruda Valder R; Tai Shing Jen; Ragni Margaret V; Thompson Arthur; Ozelo Margareth; Couto Linda B; Leonard Debra G B; Johnson Frederick A; McClelland Alan; Scallan Ciaran; Skarsgard Erik; Flake Alan W; Kay Mark A; High Katherine A; Glader Bertil
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Blood 2003;101(8):2963-72.
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2003: Ehrhardt Anja; Peng Peter D; Xu Hui; Meuse Leonard; Kay Mark A
Optimization of cis-acting elements for gene expression from nonviral vectors in vivo.
Human gene therapy 2003;14(3):215-25.
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2003: Nakai Hiroyuki; Fuess Sally; Storm Theresa A; Meuse Leonard A; Kay Mark A
Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;7(1):112-21.
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2003: Nakai Hiroyuki; Montini Eugenio; Fuess Sally; Storm Theresa A; Meuse Leonard; Finegold Milton; Grompe Markus; Kay Mark A
Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;7(1):101-11.
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2003: Sclimenti Christopher R; Neviaser Andrew S; Baba Edward J; Meuse Leonard; Kay Mark A; Calos Michele P
Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice.
Biotechnology progress 2003;19(1):144-51.
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2002: Montini Eugenio; Held Patrice K; Noll Meenakshi; Morcinek Nicolas; Al-Dhalimy Muhsen; Finegold Milton; Yant Stephen R; Kay Mark A; Grompe Markus
In vivo correction of murine tyrosinemia type I by DNA-mediated transposition.
Molecular therapy : the journal of the American Society of Gene Therapy 2002;6(6):759-69.
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2002: McCaffrey Anton P; Kay Mark A
A story of mice and men.
Gene therapy 2002;9(23):1563.
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2002: Nakai Hiroyuki; Thomas Clare E; Storm Theresa A; Fuess Sally; Powell Sharon; Wright J Fraser; Kay Mark A
A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction.
Journal of virology 2002;76(22):11343-9.
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2002: Olivares Eric C; Hollis Roger P; Chalberg Thomas W; Meuse Leonard; Kay Mark A; Calos Michele P
Site-specific genomic integration produces therapeutic Factor IX levels in mice.
Nature biotechnology 2002;20(11):1124-8.
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2002: Yant Stephen R; Ehrhardt Anja; Mikkelsen Jacob Giehm; Meuse Leonard; Pham Thao; Kay Mark A
Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo.
Nature biotechnology 2002;20(10):999-1005.
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2002: Bordier Bruno B; Marion Patricia L; Ohashi Kazuo; Kay Mark A; Greenberg Harry B; Casey John L; Glenn Jeffrey S
A prenylation inhibitor prevents production of infectious hepatitis delta virus particles.
Journal of virology 2002;76(20):10465-72.
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2002: McCaffrey Anton P; Meuse Leonard; Pham Thu-Thao T; Conklin Douglas S; Hannon Gregory J; Kay Mark A
RNA interference in adult mice.
Nature 2002;418(6893):38-9.
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2002: McCaffrey Anton P; Ohashi Kazuo; Meuse Leonard; Shen Shiliang; Lancaster Alissa M; Lukavsky Peter J; Sarnow Peter; Kay Mark A
Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2002;5(6):676-84.
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2002: Ehrhardt Anja; Kay Mark A
A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo.
Blood 2002;99(11):3923-30.
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2002: Ohashi Kazuo; Park Frank; Kay Mark A
Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo.
Human gene therapy 2002;13(5):653-63.
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2001: Arruda V R; Fields P A; Milner R; Wainwright L; De Miguel M P; Donovan P J; Herzog R W; Nichols T C; Biegel J A; Razavi M; Dake M; Huff D; Flake A W; Couto L; Kay M A; High K A
Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;4(6):586-92.
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2001: Ohashi K; Park F; Kay M A
Hepatocyte transplantation: clinical and experimental application.
Journal of molecular medicine (Berlin, Germany) 2001;79(11):617-30.
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2001: Vollrath D; Feng W; Duncan J L; Yasumura D; D'Cruz P M; Chappelow A; Matthes M T; Kay M A; LaVail M M
Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk.
Proceedings of the National Academy of Sciences of the United States of America 2001;98(22):12584-9.
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2001: Park F; Kay M A
Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;4(3):164-73.
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2001: Stoll S M; Sclimenti C R; Baba E J; Meuse L; Kay M A; Calos M P
Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;4(2):122-9.
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2001: Nakai H; Yant S R; Storm T A; Fuess S; Meuse L; Kay M A
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo.
Journal of virology 2001;75(15):6969-76.
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2001: Chen Z Y; Yant S R; He C Y; Meuse L; Shen S; Kay M A
Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;3(3):403-10.
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2001: Ohashi K; Meuse L; Schwall R; Kay M A
cMet activation allows persistent engraftment of ectopically transplanted xenogenic human hepatocytes in mice.
Transplantation proceedings 2001;33(1-2):587-8.
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2001: Kay M A; Glorioso J C; Naldini L
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics.
Nature medicine 2001;7(1):33-40.
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2000: Nakai H; Storm T A; Kay M A
Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo.
Journal of virology 2000;74(20):9451-63.
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2000: Park F; Ohashi K; Kay M A
Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver.
Blood 2000;96(3):1173-6.
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2000: Miao C H; Ohashi K; Patijn G A; Meuse L; Ye X; Thompson A R; Kay M A
Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro.
Molecular therapy : the journal of the American Society of Gene Therapy 2000;1(6):522-32.
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2000: Yant S R; Meuse L; Chiu W; Ivics Z; Izsvak Z; Kay M A
Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system.
Nature genetics 2000;25(1):35-41.
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2000: Nakai H; Storm T A; Kay M A
Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors.
Nature biotechnology 2000;18(5):527-32.
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2000: Miao C H; Nakai H; Thompson A R; Storm T A; Chiu W; Snyder R O; Kay M A
Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction.
Journal of virology 2000;74(8):3793-803.
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2000: Ohashi K; Marion P L; Nakai H; Meuse L; Cullen J M; Bordier B B; Schwall R; Greenberg H B; Glenn J S; Kay M A
Sustained survival of human hepatocytes in mice: A model for in vivo infection with human hepatitis B and hepatitis delta viruses.
Nature medicine 2000;6(3):327-31.
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2000: Kay M A; Manno C S; Ragni M V; Larson P J; Couto L B; McClelland A; Glader B; Chew A J; Tai S J; Herzog R W; Arruda V; Johnson F; Scallan C; Skarsgard E; Flake A W; High K A
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.
Nature genetics 2000;24(3):257-61.
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2000: Lieber A; Kay M A; Li Z Y
Nuclear import of moloney murine leukemia virus DNA mediated by adenovirus preterminal protein is not sufficient for efficient retroviral transduction in nondividing cells.
Journal of virology 2000;74(2):721-34.
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2000: Park F; Ohashi K; Chiu W; Naldini L; Kay M A
Efficient lentiviral transduction of liver requires cell cycling in vivo.
Nature genetics 2000;24(1):49-52.
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1999: Lieber A; Steinwaerder D S; Carlson C A; Kay M A
Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes.
Journal of virology 1999;73(11):9314-24.
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1999: Zen K; Karsan A; Stempien-Otero A; Yee E; Tupper J; Li X; Eunson T; Kay M A; Wilson C B; Winn R K; Harlan J M
NF-kappaB activation is required for human endothelial survival during exposure to tumor necrosis factor-alpha but not to interleukin-1beta or lipopolysaccharide.
The Journal of biological chemistry 1999;274(40):28808-15.
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1999: Russell D W; Kay M A
Adeno-associated virus vectors and hematology.
Blood 1999;94(3):864-74.
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1999: Nakai H; Iwaki Y; Kay M A; Couto L B
Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver.
Journal of virology 1999;73(7):5438-47.
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1999: Schowalter D B; Himeda C L; Winther B L; Wilson C B; Kay M A
Implication of interfering antibody formation and apoptosis as two different mechanisms leading to variable duration of adenovirus-mediated transgene expression in immune-competent mice.
Journal of virology 1999;73(6):4755-66.
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1999: Snyder R O; Miao C; Meuse L; Tubb J; Donahue B A; Lin H F; Stafford D W; Patel S; Thompson A R; Nichols T; Read M S; Bellinger D A; Brinkhous K M; Kay M A
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors.
Nature medicine 1999;5(1):64-70.
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1999: Patijn G A; Kay M A
Hepatic gene therapy using adeno-associated virus vectors.
Seminars in liver disease 1999;19(1):61-9.
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1998: Mizuguchi H; Kay M A
Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method.
Human gene therapy 1998;9(17):2577-83.
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1998: Lieber A; He C Y; Meuse L; Himeda C; Wilson C; Kay M A
Inhibition of NF-kappaB activation in combination with bcl-2 expression allows for persistence of first-generation adenovirus vectors in the mouse liver.
Journal of virology 1998;72(11):9267-77.
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1998: Patijn G A; Lieber A; Schowalter D B; Schwall R; Kay M A
Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice.
Hepatology (Baltimore, Md.) 1998;28(3):707-16.
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1998: Patijn G A; Terpstra O T; Kay M A
Method for continuous infusion into the portal vein of mice.
Laboratory animal science 1998;48(4):379-83.
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1998: Kay M A
Hepatic gene therapy for haemophilia B.
Haemophilia : the official journal of the World Federation of Hemophilia 1998;4(4):389-92.
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1998: Patijn G A; Lieber A; Meuse L; Winther B; Kay M A
High-efficiency retrovirus-mediated gene transfer into the livers of mice.
Human gene therapy 1998;9(10):1449-56.
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1998: Miao C H; Snyder R O; Schowalter D B; Patijn G A; Donahue B; Winther B; Kay M A
The kinetics of rAAV integration in the liver.
Nature genetics 1998;19(1):13-5.
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1997: Lieber A; He C Y; Kay M A
Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo.
Nature biotechnology 1997;15(13):1383-7.
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1997: Kay M A; Liu D; Hoogerbrugge P M
Gene therapy.
Proceedings of the National Academy of Sciences of the United States of America 1997;94(24):12744-6.
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1997: Nelson J E; Kay M A
Persistence of recombinant adenovirus in vivo is not dependent on vector DNA replication.
Journal of virology 1997;71(11):8902-7.
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1997: Lieber A; He C Y; Meuse L; Schowalter D; Kirillova I; Winther B; Kay M A
The role of Kupffer cell activation and viral gene expression in early liver toxicity after infusion of recombinant adenovirus vectors.
Journal of virology 1997;71(11):8798-807.
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1997: Schowalter D B; Kay M A
Gene therapy: a status report.
Pediatric annals 1997;26(9):562-8.
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1997: Schowalter D B; Meuse L; Wilson C B; Linsley P S; Kay M A
Constitutive expression of murine CTLA4Ig from a recombinant adenovirus vector results in prolonged transgene expression.
Gene therapy 1997;4(8):853-60.
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1997: Snyder R O; Miao C H; Patijn G A; Spratt S K; Danos O; Nagy D; Gown A M; Winther B; Meuse L; Cohen L K; Thompson A R; Kay M A
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors.
Nature genetics 1997;16(3):270-6.
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1997: Kay M A
Adenoviral vectors for hepatic gene transfer in animals.
Chest 1997;111(6 Suppl):138S-142S.
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1997: Schowalter D B; Tubb J C; Liu M; Wilson C B; Kay M A
Heterologous expression of adenovirus E3-gp19K in an E1a-deleted adenovirus vector inhibits MHC I expression in vitro, but does not prolong transgene expression in vivo.
Gene therapy 1997;4(4):351-60.
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1997: Kay M A; Meuse L; Gown A M; Linsley P; Hollenbaugh D; Aruffo A; Ochs H D; Wilson C B
Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver.
Proceedings of the National Academy of Sciences of the United States of America 1997;94(9):4686-91.
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1997: Vrancken Peeters M J; Patijn G A; Lieber A; Perkins J; Kay M A
Expansion of donor hepatocytes after recombinant adenovirus-induced liver regeneration in mice.
Hepatology (Baltimore, Md.) 1997;25(4):884-8.
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1997: Kay M A; Fausto N
Liver regeneration: prospects for therapy based on new technologies.
Molecular medicine today 1997;3(3):108-15.
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1996: Lieber A; He C Y; Kirillova I; Kay M A
Recombinant adenoviruses with large deletions generated by Cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo.
Journal of virology 1996;70(12):8944-60.
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1996: Lieber A; He C Y; Polyak S J; Gretch D R; Barr D; Kay M A
Elimination of hepatitis C virus RNA in infected human hepatocytes by adenovirus-mediated expression of ribozymes.
Journal of virology 1996;70(12):8782-91.
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1996: Thompson A R; Kay M A
Nonviral gene transfer to the liver.
Hepatology (Baltimore, Md.) 1996;24(6):1541-2.
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1996: Sandig V; Löser P; Lieber A; Kay M A; Strauss M
HBV-derived promoters direct liver-specific expression of an adenovirally transduced LDL receptor gene.
Gene therapy 1996;3(11):1002-9.
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1996: Peeters M J; Patijn G A; Lieber A; Meuse L; Kay M A
Adenovirus-mediated hepatic gene transfer in mice: comparison of intravascular and biliary administration.
Human gene therapy 1996;7(14):1693-9.
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1996: Lieber A; Kay M A
Adenovirus-mediated expression of ribozymes in mice.
Journal of virology 1996;70(5):3153-8.
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1996: Liu M L; Winther B L; Kay M A
Pseudotransduction of hepatocytes by using concentrated pseudotyped vesicular stomatitis virus G glycoprotein (VSV-G)-Moloney murine leukemia virus-derived retrovirus vectors: comparison of VSV-G and amphotropic vectors for hepatic gene transfer.
Journal of virology 1996;70(4):2497-502.
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1996: Vrancken Peeters M J; Perkins A L; Kay M A
Method for multiple portal vein infusions in mice: quantitation of adenovirus-mediated hepatic gene transfer.
BioTechniques 1996;20(2):278-85.
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1995: Kay M A; Holterman A X; Meuse L; Gown A; Ochs H D; Linsley P S; Wilson C B
Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration.
Nature genetics 1995;11(2):191-7.
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1995: Wilson C; Kay M A
Immunomodulation to enhance gene therapy.
Nature medicine 1995;1(9):887-9.
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1995: Fang B; Eisensmith R C; Wang H; Kay M A; Cross R E; Landen C N; Gordon G; Bellinger D A; Read M S; Hu P C
Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression.
Human gene therapy 1995;6(8):1039-44.
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1995: Lieber A; Peeters M J; Gown A; Perkins J; Kay M A
A modified urokinase plasminogen activator induces liver regeneration without bleeding.
Human gene therapy 1995;6(8):1029-37.
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1995: Lieber A; Vrancken Peeters M J; Meuse L; Fausto N; Perkins J; Kay M A
Adenovirus-mediated urokinase gene transfer induces liver regeneration and allows for efficient retrovirus transduction of hepatocytes in vivo.
Proceedings of the National Academy of Sciences of the United States of America 1995;92(13):6210-4.
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1995: Scaria A; Curiel D T; Kay M A
Complementation of a human adenovirus early region 4 deletion mutant in 293 cells using adenovirus-polylysine-DNA complexes.
Gene therapy 1995;2(4):295-8.
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1995: Kay M A; Graham F; Leland F; Woo S L
Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes.
Hepatology (Baltimore, Md.) 1995;21(3):815-9.
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1995: Barr D; Tubb J; Ferguson D; Scaria A; Lieber A; Wilson C; Perkins J; Kay M A
Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains.
Gene therapy 1995;2(2):151-5.
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1995: Lieber A; Vrancken Peeters M J; Kay M A
Adenovirus-mediated transfer of the amphotropic retrovirus receptor cDNA increases retroviral transduction in cultured cells.
Human gene therapy 1995;6(1):5-11.
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1995: Kay M A
Hepatic gene therapy for hemophilia B.
Advances in experimental medicine and biology 1995;386():229-34.
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1994: Kay M A; Landen C N; Rothenberg S R; Taylor L A; Leland F; Wiehle S; Fang B; Bellinger D; Finegold M; Thompson A R
In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.
Proceedings of the National Academy of Sciences of the United States of America 1994;91(6):2353-7.
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1993: Cristiano R J; Smith L C; Kay M A; Brinkley B R; Woo S L
Hepatic gene therapy: efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex.
Proceedings of the National Academy of Sciences of the United States of America 1993;90(24):11548-52.
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1993: Kay M A; Rothenberg S; Landen C N; Bellinger D A; Leland F; Toman C; Finegold M; Thompson A R; Read M S; Brinkhous K M
In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs.
Science (New York, N.Y.) 1993;262(5130):117-9.
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1993: Kolodka T M; Finegold M; Kay M A; Woo S L
Hepatic gene therapy: efficient retroviral-mediated gene transfer into rat hepatocytes in vivo.
Somatic cell and molecular genetics 1993;19(5):491-7.
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1993: Li Q; Kay M A; Finegold M; Stratford-Perricaudet L D; Woo S L
Assessment of recombinant adenoviral vectors for hepatic gene therapy.
Human gene therapy 1993;4(4):403-9.
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1992: Kay M A; Li Q; Liu T J; Leland F; Toman C; Finegold M; Woo S L
Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo.
Human gene therapy 1992;3(6):641-7.
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1992: Kay M A; Baley P; Rothenberg S; Leland F; Fleming L; Ponder K P; Liu T; Finegold M; Darlington G; Pokorny W
Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes.
Proceedings of the National Academy of Sciences of the United States of America 1992;89(1):89-93.
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1992: Liu T J; Kay M A; Darlington G J; Woo S L
Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice.
Somatic cell and molecular genetics 1992;18(1):89-96.
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1992: Kay M A; Ponder K P; Woo S L
Human gene therapy: present and future.
Breast cancer research and treatment 1992;21(2):83-93.
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