Mark A. Kay, M.D., Ph.D.
Publications
- A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro. Mol Ther. 2013; (5): 954-63
- Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther. 2013; (1): 131-8
- The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA. Nucleic Acids Res. 2013; (6): 3688-98
- Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucleic Acids Res. 2012; (8): 3704-13
- Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circ Res. 2012; (12): 1494-503
- Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Mol Ther. 2012; (10): 1912-23
- Slicing-independent RISC activation requires the argonaute PAZ domain. Curr Biol. 2012; (16): 1536-42
- The extragenic spacer length between the 5' and 3' ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors. Mol Ther. 2012; (11): 2111-9
- The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo. Cell. 2012; (4): 900-11
- rAAV-mediated tumorigenesis: still unresolved after an AAV assault. Mol Ther. 2012; (11): 2014-7
- Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011; (25): 2357-65
- Double knockdown of prolyl hydroxylase and factor-inhibiting hypoxia-inducible factor with nonviral minicircle gene therapy enhances stem cell mobilization and angiogenesis after myocardial infarction. Circulation. 2011; (11 Suppl): S46-54
- Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Nat Protoc. 2011; (1): 78-88
- State-of-the-art gene-based therapies: the road ahead. Nat Rev Genet. 2011; (5): 316-28
- Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes. Proc Natl Acad Sci U S A. 2011; (22): 9208-13
- A nonviral minicircle vector for deriving human iPS cells. Nat Methods. 2010; (3): 197-9
- A robust system for production of minicircle DNA vectors. Nat Biotechnol. 2010; (12): 1287-9
- An in vitro-identified high-affinity nucleosome-positioning signal is capable of transiently positioning a nucleosome in vivo. Epigenetics Chromatin. 2010; (1): 13
- Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest. 2010; (9): 3106-19
- How do miRNAs mediate translational repression? Silence. 2010; (1): 11
- Human tRNA-derived small RNAs in the global regulation of RNA silencing. RNA. 2010; (4): 673-95
- Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice. Mol Ther. 2010; (1): 161-70
- Six RNA viruses and forty-one hosts: viral small RNAs and modulation of small RNA repertoires in vertebrate and invertebrate systems. PLoS Pathog. 2010; (2): e1000764
- miR-122 continues to blaze the trail for microRNA therapeutics. Mol Ther. 2010; (2): 240-2
- Biological basis for restriction of microRNA targets to the 3' untranslated region in mammalian mRNAs. Nat Struct Mol Biol. 2009; (2): 144-50
- Combined proteomic-RNAi screen for host factors involved in human hepatitis delta virus replication. RNA. 2009; (11): 1971-9
- Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor. J Cardiovasc Pharmacol. 2009; (1): 18-23
- Novel minicircle vector for gene therapy in murine myocardial infarction. Circulation. 2009; (11 Suppl): S230-7
- Capped small RNAs and MOV10 in human hepatitis delta virus replication. Nat Struct Mol Biol. 2008; (7): 714-21
- Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Mol Ther. 2008; (9): 1630-6
- Hepatic parenchymal replacement in mice by transplanted allogeneic hepatocytes is facilitated by bone marrow transplantation and mediated by CD4 cells. Hepatology. 2008; (2): 706-18
- In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol. 2008; (12): 5887-911
- Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Mol Ther. 2008; (3): 548-56
- A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8. Mol Ther. 2007; (2): 330-9
- AAV vectors and tumorigenicity. Nat Biotechnol. 2007; (10): 1111-3
- Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver. Mol Ther. 2007; (2): 255-63
- Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency. Mol Ther. 2007; (11): 1955-62
- Combinatorial RNAi: a winning strategy for the race against evolving targets? Mol Ther. 2007; (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. J Virol. 2007; (20): 11290-303
- Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo. Mol Ther. 2007; (7): 1348-55
- Postintegrative gene silencing within the Sleeping Beauty transposition system. Mol Cell Biol. 2007; (24): 8824-33
- RNAi and gene therapy: a mutual attraction. Hematology Am Soc Hematol Educ Program. 2007: 473-81
- Site-directed transposon integration in human cells. Nucleic Acids Res. 2007; (7): e50
- Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo. Mol Ther. 2007; (1): 146-56
- The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. J Virol. 2007; (20): 11304-21
- Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? J Clin Invest. 2007; (12): 3633-41
- Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature. 2006; (7092): 537-41
- Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J Virol. 2006; (1): 426-39
- Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration. Hum Gene Ther. 2006; (11): 1077-94
- The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J Virol. 2006; (19): 9831-6
- Therapeutic short hairpin RNA expression in the liver: viral targets and vectors. Gene Ther. 2006; (6): 563-75
- A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase. Mol Ther. 2005; (5): 695-706
- Adeno-associated virus vectors for short hairpin RNA expression. Methods Enzymol. 2005: 381-405
- Genomic progression in mouse models for liver tumors. Cold Spring Harb Symp Quant Biol. 2005: 217-24
- High-resolution genome-wide mapping of transposon integration in mammals. Mol Cell Biol. 2005; (6): 2085-94
- Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo. Hum Gene Ther. 2005; (1): 126-31
- Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo. Hum Gene Ther. 2005; (5): 558-70
- Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. J Virol. 2005; (6): 3606-14
- Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases. Hepatology. 2005; (1): 132-40
- Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver. Hum Gene Ther. 2005; (3): 299-306
- Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol. 2005; (1): 214-24
- Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo. Hum Gene Ther. 2004; (4): 405-13
- Donor-derived, liver-specific protein expression after bone marrow transplantation. Transplantation. 2004; (4): 530-6
- Efficient inhibition of in-stent restenosis by controlled stent-based inhibition of elastase: a pilot study. J Vasc Interv Radiol. 2004; (11): 1287-93
- Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice. Transplant Proc. 2004; (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. Mol Cell Biol. 2004; (20): 9239-47
- Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol. 2004; (6): 3110-22
- Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Ther. 2004; (10): 856-64
- Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice. Biotechnol Prog. 2003 Jan-Feb; (1): 144-51
- A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood. 2003; (7): 2403-11
- A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice. Hepatology. 2003; (2): 503-8
- AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet. 2003; (3): 297-302
- Advancing molecular therapies through in vivo bioluminescent imaging. Mol Imaging. 2003; (2): 75-86
- Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo. J Virol. 2003; (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. Mol Ther. 2003; (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. Curr Gene Ther. 2003; (4): 281-304
- Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther. 2003; (6): 839-50
- Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol Ther. 2003; (4): 654-65
- Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice. Mol Ther. 2003; (1): 101-11
- In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus. J Clin Invest. 2003; (3): 407-14
- Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol. 2003; (6): 639-44
- Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther. 2003; (3): 495-500
- Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells. Mol Cell Biol. 2003; (23): 8505-18
- Optimization of cis-acting elements for gene expression from nonviral vectors in vivo. Hum Gene Ther. 2003; (3): 215-25
- Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors. Hum Gene Ther. 2003; (9): 871-81
- Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood. 2003; (7): 2412-9
- Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet. 2003; (5): 346-58
- Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer. Gene Ther. 2003; (13): 1099-104
- System for simultaneous tissue-specific and disease-specific regulation of therapeutic gene expression. Hum Gene Ther. 2003; (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. J Virol. 2002; (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. Blood. 2002; (11): 3923-30
- A prenylation inhibitor prevents production of infectious hepatitis delta virus particles. J Virol. 2002; (20): 10465-72
- A story of mice and men. Gene Ther. 2002; (23): 1563
- Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice. Mol Ther. 2002; (6): 676-84
- Efficient gene transduction to cultured hepatocytes by HIV-1 derived lentiviral vector. Transplant Proc. 2002; (5): 1431-3
- RNA interference in adult mice. Nature. 2002; (6893): 38-9
- Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo. Hum Gene Ther. 2002; (5): 653-63
- Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol. 2002; (11): 1124-8
- Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat Biotechnol. 2002; (10): 999-1005
- cMet activation allows persistent engraftment of ectopically transplanted xenogenic human hepatocytes in mice. Transplant Proc. 2001 Feb-Mar; (1-2): 587-8
- Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc Natl Acad Sci U S A. 2001; (22): 12584-9
- Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice. Mol Ther. 2001; (2): 122-9
- Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol. 2001; (15): 6969-76
- Hepatocyte transplantation: clinical and experimental application. J Mol Med (Berl). 2001; (11): 617-30
- Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. Mol Ther. 2001; (3): 403-10
- Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo. Mol Ther. 2001; (3): 164-73
- Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med. 2001; (1): 33-40
- Efficient lentiviral transduction of liver requires cell cycling in vivo. Nat Genet. 2000; (1): 49-52
- Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet. 2000; (3): 257-61
- Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat Biotechnol. 2000; (5): 527-32
- Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J Virol. 2000; (20): 9451-63
- Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat Genet. 2000; (1): 35-41
- Sustained survival of human hepatocytes in mice: A model for in vivo infection with human hepatitis B and hepatitis delta viruses. Nat Med. 2000; (3): 327-31
- Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood. 2000; (3): 1173-6
- Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med. 1999; (1): 64-70
- Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. J Virol. 1999; (7): 5438-47
- Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method. Hum Gene Ther. 1998; (17): 2577-83
- Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo. Nat Biotechnol. 1997; (13): 1383-7
- In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science. 1993; (5130): 117-9
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