Publications
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A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro.
Lu J,
Zhang F, Kay MA.
Mol Ther.
2013;
21
(5):
954-63
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Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level.
Gracey Maniar LE,
Maniar JM, Chen ZY, Lu J, Fire AZ, Kay MA.
Mol Ther.
2013;
21
(1):
131-8
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The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA.
Lisowski L,
Elazar M, Chu K, Glenn JS, Kay MA.
Nucleic Acids Res.
2013;
41
(6):
3688-98
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Expression determinants of mammalian argonaute proteins in mediating gene silencing.
Valdmanis PN,
Gu S, Schüermann N, Sethupathy P, Grimm D, Kay MA.
Nucleic Acids Res.
2012;
40
(8):
3704-13
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Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging.
Wang Y,
Zhang WY, Hu S, Lan F, Lee AS, Huber B, Lisowski L, Liang P, Huang M, de Almeida PE, Won JH, Sun N, Robbins RC, Kay MA, Urnov FD, Wu JC.
Circ Res.
2012;
111
(12):
1494-503
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Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression.
Lisowski L,
Lau A, Wang Z, Zhang Y, Zhang F, Grompe M, Kay MA.
Mol Ther.
2012;
20
(10):
1912-23
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Slicing-independent RISC activation requires the argonaute PAZ domain.
Gu S,
Jin L, Huang Y, Zhang F, Kay MA.
Curr Biol.
2012;
22
(16):
1536-42
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The extragenic spacer length between the 5' and 3' ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors.
Lu J,
Zhang F, Xu S, Fire AZ, Kay MA.
Mol Ther.
2012;
20
(11):
2111-9
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The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo.
Gu S,
Jin L, Zhang Y, Huang Y, Zhang F, Valdmanis PN, Kay MA.
Cell.
2012;
151
(4):
900-11
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rAAV-mediated tumorigenesis: still unresolved after an AAV assault.
Valdmanis PN,
Lisowski L, Kay MA.
Mol Ther.
2012;
20
(11):
2014-7
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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Nathwani AC,
Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM.
N Engl J Med.
2011;
365
(25):
2357-65
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Double knockdown of prolyl hydroxylase and factor-inhibiting hypoxia-inducible factor with nonviral minicircle gene therapy enhances stem cell mobilization and angiogenesis after myocardial infarction.
Huang M,
Nguyen P, Jia F, Hu S, Gong Y, de Almeida PE, Wang L, Nag D, Kay MA, Giaccia AJ, Robbins RC, Wu JC.
Circulation.
2011;
124
(11 Suppl):
S46-54
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Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors.
Narsinh KH,
Jia F, Robbins RC, Kay MA, Longaker MT, Wu JC.
Nat Protoc.
2011;
6
(1):
78-88
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State-of-the-art gene-based therapies: the road ahead.
Kay MA,
Nat Rev Genet.
2011;
12
(5):
316-28
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Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes.
Gu S,
Jin L, Zhang F, Huang Y, Grimm D, Rossi JJ, Kay MA.
Proc Natl Acad Sci U S A.
2011;
108
(22):
9208-13
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A nonviral minicircle vector for deriving human iPS cells.
Jia F,
Wilson KD, Sun N, Gupta DM, Huang M, Li Z, Panetta NJ, Chen ZY, Robbins RC, Kay MA, Longaker MT, Wu JC.
Nat Methods.
2010;
7
(3):
197-9
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A robust system for production of minicircle DNA vectors.
Kay MA,
He CY, Chen ZY.
Nat Biotechnol.
2010;
28
(12):
1287-9
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An in vitro-identified high-affinity nucleosome-positioning signal is capable of transiently positioning a nucleosome in vivo.
Gracey LE,
Chen ZY, Maniar JM, Valouev A, Sidow A, Kay MA, Fire AZ.
Epigenetics Chromatin.
2010;
3
(1):
13
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Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver.
Grimm D,
Wang L, Lee JS, Schürmann N, Gu S, Börner K, Storm TA, Kay MA.
J Clin Invest.
2010;
120
(9):
3106-19
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How do miRNAs mediate translational repression?
Gu S,
Kay MA.
Silence.
2010;
1
(1):
11
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Human tRNA-derived small RNAs in the global regulation of RNA silencing.
Haussecker D,
Huang Y, Lau A, Parameswaran P, Fire AZ, Kay MA.
RNA.
2010;
16
(4):
673-95
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Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice.
Beer S,
Bellovin DI, Lee JS, Komatsubara K, Wang LS, Koh H, Börner K, Storm TA, Davis CR, Kay MA, Felsher DW, Grimm D.
Mol Ther.
2010;
18
(1):
161-70
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Six RNA viruses and forty-one hosts: viral small RNAs and modulation of small RNA repertoires in vertebrate and invertebrate systems.
Parameswaran P,
Sklan E, Wilkins C, Burgon T, Samuel MA, Lu R, Ansel KM, Heissmeyer V, Einav S, Jackson W, Doukas T, Paranjape S, Polacek C, dos Santos FB, Jalili R, Babrzadeh F, Gharizadeh B, Grimm D, Kay M, Koike S, Sarnow P, Ronaghi M, Ding SW, Harris E, Chow M, Diamond MS, Kirkegaard K, Glenn JS, Fire AZ.
PLoS Pathog.
2010;
6
(2):
e1000764
-
miR-122 continues to blaze the trail for microRNA therapeutics.
Haussecker D,
Kay MA.
Mol Ther.
2010;
18
(2):
240-2
-
Biological basis for restriction of microRNA targets to the 3' untranslated region in mammalian mRNAs.
Gu S,
Jin L, Zhang F, Sarnow P, Kay MA.
Nat Struct Mol Biol.
2009;
16
(2):
144-50
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Combined proteomic-RNAi screen for host factors involved in human hepatitis delta virus replication.
Cao D,
Haussecker D, Huang Y, Kay MA.
RNA.
2009;
15
(11):
1971-9
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Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor.
Stenler S,
Andersson A, Simonson OE, Lundin KE, Chen ZY, Kay MA, Smith CI, Sylvén C, Blomberg P.
J Cardiovasc Pharmacol.
2009;
53
(1):
18-23
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Novel minicircle vector for gene therapy in murine myocardial infarction.
Huang M,
Chen Z, Hu S, Jia F, Li Z, Hoyt G, Robbins RC, Kay MA, Wu JC.
Circulation.
2009;
120
(11 Suppl):
S230-7
-
Capped small RNAs and MOV10 in human hepatitis delta virus replication.
Haussecker D,
Cao D, Huang Y, Parameswaran P, Fire AZ, Kay MA.
Nat Struct Mol Biol.
2008;
15
(7):
714-21
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Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic.
Giering JC,
Grimm D, Storm TA, Kay MA.
Mol Ther.
2008;
16
(9):
1630-6
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Hepatic parenchymal replacement in mice by transplanted allogeneic hepatocytes is facilitated by bone marrow transplantation and mediated by CD4 cells.
Streetz KL,
Doyonnas R, Grimm D, Jenkins DD, Fuess S, Perryman S, Lin J, Trautwein C, Shizuru J, Blau H, Sylvester KG, Kay MA.
Hepatology.
2008;
47
(2):
706-18
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In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses.
Grimm D,
Lee JS, Wang L, Desai T, Akache B, Storm TA, Kay MA.
J Virol.
2008;
82
(12):
5887-911
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Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation.
Chen ZY,
Riu E, He CY, Xu H, Kay MA.
Mol Ther.
2008;
16
(3):
548-56
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A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8.
Akache B,
Grimm D, Shen X, Fuess S, Yant SR, Glazer DS, Park J, Kay MA.
Mol Ther.
2007;
15
(2):
330-9
-
AAV vectors and tumorigenicity.
Kay MA,
Nat Biotechnol.
2007;
25
(10):
1111-3
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Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver.
Wang AY,
Ehrhardt A, Xu H, Kay MA.
Mol Ther.
2007;
15
(2):
255-63
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Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency.
Shen X,
Storm T, Kay MA.
Mol Ther.
2007;
15
(11):
1955-62
-
Combinatorial RNAi: a winning strategy for the race against evolving targets?
Grimm D,
Kay MA.
Mol Ther.
2007;
15
(5):
878-88
-
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.
Inagaki K,
Lewis SM, Wu X, Ma C, Munroe DJ, Fuess S, Storm TA, Kay MA, Nakai H.
J Virol.
2007;
81
(20):
11290-303
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Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo.
Riu E,
Chen ZY, Xu H, He CY, Kay MA.
Mol Ther.
2007;
15
(7):
1348-55
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Postintegrative gene silencing within the Sleeping Beauty transposition system.
Garrison BS,
Yant SR, Mikkelsen JG, Kay MA.
Mol Cell Biol.
2007;
27
(24):
8824-33
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RNAi and gene therapy: a mutual attraction.
Grimm D,
Kay MA.
Hematology Am Soc Hematol Educ Program.
2007:
473-81
-
Site-directed transposon integration in human cells.
Yant SR,
Huang Y, Akache B, Kay MA.
Nucleic Acids Res.
2007;
35
(7):
e50
-
Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo.
Ehrhardt A,
Yant SR, Giering JC, Xu H, Engler JA, Kay MA.
Mol Ther.
2007;
15
(1):
146-56
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The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice.
Inagaki K,
Ma C, Storm TA, Kay MA, Nakai H.
J Virol.
2007;
81
(20):
11304-21
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Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?
Grimm D,
Kay MA.
J Clin Invest.
2007;
117
(12):
3633-41
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Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.
Grimm D,
Streetz KL, Jopling CL, Storm TA, Pandey K, Davis CR, Marion P, Salazar F, Kay MA.
Nature.
2006;
441
(7092):
537-41
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Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype.
Grimm D,
Pandey K, Nakai H, Storm TA, Kay MA.
J Virol.
2006;
80
(1):
426-39
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Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration.
Ehrhardt A,
Engler JA, Xu H, Cherry AM, Kay MA.
Hum Gene Ther.
2006;
17
(11):
1077-94
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The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9.
Akache B,
Grimm D, Pandey K, Yant SR, Xu H, Kay MA.
J Virol.
2006;
80
(19):
9831-6
-
Therapeutic short hairpin RNA expression in the liver: viral targets and vectors.
Grimm D,
Kay MA.
Gene Ther.
2006;
13
(6):
563-75
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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.
Ehrhardt A,
Xu H, Huang Z, Engler JA, Kay MA.
Mol Ther.
2005;
11
(5):
695-706
-
Adeno-associated virus vectors for short hairpin RNA expression.
Grimm D,
Pandey K, Kay MA.
Methods Enzymol.
2005:
392
381-405
-
Genomic progression in mouse models for liver tumors.
Tward AD,
Jones KD, Yant S, Kay MA, Wang R, Bishop JM.
Cold Spring Harb Symp Quant Biol.
2005:
70
217-24
-
High-resolution genome-wide mapping of transposon integration in mammals.
Yant SR,
Wu X, Huang Y, Garrison B, Burgess SM, Kay MA.
Mol Cell Biol.
2005;
25
(6):
2085-94
-
Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo.
Chen ZY,
He CY, Kay MA.
Hum Gene Ther.
2005;
16
(1):
126-31
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Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo.
Riu E,
Grimm D, Huang Z, Kay MA.
Hum Gene Ther.
2005;
16
(5):
558-70
-
Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver.
Nakai H,
Wu X, Fuess S, Storm TA, Munroe D, Montini E, Burgess SM, Grompe M, Kay MA.
J Virol.
2005;
79
(6):
3606-14
-
Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases.
Ohashi K,
Waugh JM, Dake MD, Yokoyama T, Kuge H, Nakajima Y, Yamanouchi M, Naka H, Yoshioka A, Kay MA.
Hepatology.
2005;
41
(1):
132-40
-
Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver.
Ohashi K,
Nakai H, Couto LB, Kay MA.
Hum Gene Ther.
2005;
16
(3):
299-306
-
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice.
Nakai H,
Fuess S, Storm TA, Muramatsu S, Nara Y, Kay MA.
J Virol.
2005;
79
(1):
214-24
-
Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo.
Wang AY,
Peng PD, Ehrhardt A, Storm TA, Kay MA.
Hum Gene Ther.
2004;
15
(4):
405-13
-
Donor-derived, liver-specific protein expression after bone marrow transplantation.
Jenkins DD,
Streetz K, Tataria M, Sahar D, Kurobe M, Longaker MT, Kay MA, Sylvester KG.
Transplantation.
2004;
78
(4):
530-6
-
Efficient inhibition of in-stent restenosis by controlled stent-based inhibition of elastase: a pilot study.
Ganaha F,
Ohashi K, Do YS, Lee J, Sugimoto K, Minamiguchi H, Elkins CJ, Sameni D, Modanlou S, Ali M, Kao EY, Kay MA, Waugh JM, Dake MD.
J Vasc Interv Radiol.
2004;
15
(11):
1287-93
-
Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice.
Ohashi K,
Kay MA.
Transplant Proc.
2004;
36
(8):
2469-70
-
Mutational analysis of the N-terminal DNA-binding domain of sleeping beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells.
Yant SR,
Park J, Huang Y, Mikkelsen JG, Kay MA.
Mol Cell Biol.
2004;
24
(20):
9239-47
-
Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors.
Thomas CE,
Storm TA, Huang Z, Kay MA.
J Virol.
2004;
78
(6):
3110-22
-
Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo.
Chen ZY,
He CY, Meuse L, Kay MA.
Gene Ther.
2004;
11
(10):
856-64
-
Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice.
Sclimenti CR,
Neviaser AS, Baba EJ, Meuse L, Kay MA, Calos MP.
Biotechnol Prog.
2003 Jan-Feb;
19
(1):
144-51
-
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia.
Ehrhardt A,
Xu H, Dillow AM, Bellinger DA, Nichols TC, Kay MA.
Blood.
2003;
102
(7):
2403-11
-
A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice.
McCaffrey AP,
Meuse L, Karimi M, Contag CH, Kay MA.
Hepatology.
2003;
38
(2):
503-8
-
AAV serotype 2 vectors preferentially integrate into active genes in mice.
Nakai H,
Montini E, Fuess S, Storm TA, Grompe M, Kay MA.
Nat Genet.
2003;
34
(3):
297-302
-
Advancing molecular therapies through in vivo bioluminescent imaging.
McCaffrey A,
Kay MA, Contag CH.
Mol Imaging.
2003;
2
(2):
75-86
-
Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo.
Ehrhardt A,
Xu H, Kay MA.
J Virol.
2003;
77
(13):
7689-95
-
Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo.
Nakai H,
Fuess S, Storm TA, Meuse LA, Kay MA.
Mol Ther.
2003;
7
(1):
112-21
-
From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy.
Grimm D,
Kay MA.
Curr Gene Ther.
2003;
3
(4):
281-304
-
Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6.
Grimm D,
Kay MA, Kleinschmidt JA.
Mol Ther.
2003;
7
(6):
839-50
-
Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo.
Mikkelsen JG,
Yant SR, Meuse L, Huang Z, Xu H, Kay MA.
Mol Ther.
2003;
8
(4):
654-65
-
Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice.
Nakai H,
Montini E, Fuess S, Storm TA, Meuse L, Finegold M, Grompe M, Kay MA.
Mol Ther.
2003;
7
(1):
101-11
-
In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus.
Bordier BB,
Ohkanda J, Liu P, Lee SY, Salazar FH, Marion PL, Ohashi K, Meuse L, Kay MA, Casey JL, Sebti SM, Hamilton AD, Glenn JS.
J Clin Invest.
2003;
112
(3):
407-14
-
Inhibition of hepatitis B virus in mice by RNA interference.
McCaffrey AP,
Nakai H, Pandey K, Huang Z, Salazar FH, Xu H, Wieland SF, Marion PL, Kay MA.
Nat Biotechnol.
2003;
21
(6):
639-44
-
Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo.
Chen ZY,
He CY, Ehrhardt A, Kay MA.
Mol Ther.
2003;
8
(3):
495-500
-
Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells.
Yant SR,
Kay MA.
Mol Cell Biol.
2003;
23
(23):
8505-18
-
Optimization of cis-acting elements for gene expression from nonviral vectors in vivo.
Ehrhardt A,
Peng PD, Xu H, Meuse L, Kay MA.
Hum Gene Ther.
2003;
14
(3):
215-25
-
Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors.
Nakai H,
Storm TA, Fuess S, Kay MA.
Hum Gene Ther.
2003;
14
(9):
871-81
-
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy.
Grimm D,
Zhou S, Nakai H, Thomas CE, Storm TA, Fuess S, Matsushita T, Allen J, Surosky R, Lochrie M, Meuse L, McClelland A, Colosi P, Kay MA.
Blood.
2003;
102
(7):
2412-9
-
Progress and problems with the use of viral vectors for gene therapy.
Thomas CE,
Ehrhardt A, Kay MA.
Nat Rev Genet.
2003;
4
(5):
346-58
-
Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer.
Ortiz-Urda S,
Lin Q, Yant SR, Keene D, Kay MA, Khavari PA.
Gene Ther.
2003;
10
(13):
1099-104
-
System for simultaneous tissue-specific and disease-specific regulation of therapeutic gene expression.
Chyung YH,
Peng PD, Kay MA.
Hum Gene Ther.
2003;
14
(13):
1255-64
-
A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction.
Nakai H,
Thomas CE, Storm TA, Fuess S, Powell S, Wright JF, Kay MA.
J Virol.
2002;
76
(22):
11343-9
-
A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo.
Ehrhardt A,
Kay MA.
Blood.
2002;
99
(11):
3923-30
-
A prenylation inhibitor prevents production of infectious hepatitis delta virus particles.
Bordier BB,
Marion PL, Ohashi K, Kay MA, Greenberg HB, Casey JL, Glenn JS.
J Virol.
2002;
76
(20):
10465-72
-
A story of mice and men.
McCaffrey AP,
Kay MA.
Gene Ther.
2002;
9
(23):
1563
-
Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice.
McCaffrey AP,
Ohashi K, Meuse L, Shen S, Lancaster AM, Lukavsky PJ, Sarnow P, Kay MA.
Mol Ther.
2002;
5
(6):
676-84
-
Efficient gene transduction to cultured hepatocytes by HIV-1 derived lentiviral vector.
Ohashi K,
Park F, Schwall R, Kay M.
Transplant Proc.
2002;
34
(5):
1431-3
-
RNA interference in adult mice.
McCaffrey AP,
Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA.
Nature.
2002;
418
(6893):
38-9
-
Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo.
Ohashi K,
Park F, Kay MA.
Hum Gene Ther.
2002;
13
(5):
653-63
-
Site-specific genomic integration produces therapeutic Factor IX levels in mice.
Olivares EC,
Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP.
Nat Biotechnol.
2002;
20
(11):
1124-8
-
Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo.
Yant SR,
Ehrhardt A, Mikkelsen JG, Meuse L, Pham T, Kay MA.
Nat Biotechnol.
2002;
20
(10):
999-1005
-
cMet activation allows persistent engraftment of ectopically transplanted xenogenic human hepatocytes in mice.
Ohashi K,
Meuse L, Schwall R, Kay MA.
Transplant Proc.
2001 Feb-Mar;
33
(1-2):
587-8
-
Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk.
Vollrath D,
Feng W, Duncan JL, Yasumura D, D'Cruz PM, Chappelow A, Matthes MT, Kay MA, LaVail MM.
Proc Natl Acad Sci U S A.
2001;
98
(22):
12584-9
-
Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice.
Stoll SM,
Sclimenti CR, Baba EJ, Meuse L, Kay MA, Calos MP.
Mol Ther.
2001;
4
(2):
122-9
-
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo.
Nakai H,
Yant SR, Storm TA, Fuess S, Meuse L, Kay MA.
J Virol.
2001;
75
(15):
6969-76
-
Hepatocyte transplantation: clinical and experimental application.
Ohashi K,
Park F, Kay MA.
J Mol Med (Berl).
2001;
79
(11):
617-30
-
Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver.
Chen ZY,
Yant SR, He CY, Meuse L, Shen S, Kay MA.
Mol Ther.
2001;
3
(3):
403-10
-
Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo.
Park F,
Kay MA.
Mol Ther.
2001;
4
(3):
164-73
-
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics.
Kay MA,
Glorioso JC, Naldini L.
Nat Med.
2001;
7
(1):
33-40
-
Efficient lentiviral transduction of liver requires cell cycling in vivo.
Park F,
Ohashi K, Chiu W, Naldini L, Kay MA.
Nat Genet.
2000;
24
(1):
49-52
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Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.
Kay MA,
Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA.
Nat Genet.
2000;
24
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257-61
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Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors.
Nakai H,
Storm TA, Kay MA.
Nat Biotechnol.
2000;
18
(5):
527-32
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Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo.
Nakai H,
Storm TA, Kay MA.
J Virol.
2000;
74
(20):
9451-63
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Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system.
Yant SR,
Meuse L, Chiu W, Ivics Z, Izsvak Z, Kay MA.
Nat Genet.
2000;
25
(1):
35-41
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Sustained survival of human hepatocytes in mice: A model for in vivo infection with human hepatitis B and hepatitis delta viruses.
Ohashi K,
Marion PL, Nakai H, Meuse L, Cullen JM, Bordier BB, Schwall R, Greenberg HB, Glenn JS, Kay MA.
Nat Med.
2000;
6
(3):
327-31
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Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver.
Park F,
Ohashi K, Kay MA.
Blood.
2000;
96
(3):
1173-6
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Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors.
Snyder RO,
Miao C, Meuse L, Tubb J, Donahue BA, Lin HF, Stafford DW, Patel S, Thompson AR, Nichols T, Read MS, Bellinger DA, Brinkhous KM, Kay MA.
Nat Med.
1999;
5
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64-70
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Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver.
Nakai H,
Iwaki Y, Kay MA, Couto LB.
J Virol.
1999;
73
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5438-47
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Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method.
Mizuguchi H,
Kay MA.
Hum Gene Ther.
1998;
9
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2577-83
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Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo.
Lieber A,
He CY, Kay MA.
Nat Biotechnol.
1997;
15
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1383-7
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In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs.
Kay MA,
Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM.
Science.
1993;
262
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117-9
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