Mark A Kay

Author PubWeight™ 226.75‹?›

Top papers

Rank Title Journal Year PubWeight™‹?›
1 Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006 13.51
2 Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011 11.01
3 Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006 10.95
4 Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 2003 6.95
5 RNA interference in adult mice. Nature 2002 5.33
6 Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice. Nat Biotechnol 2007 5.04
7 Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther 2006 4.64
8 AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2002 4.60
9 A nonviral minicircle vector for deriving human iPS cells. Nat Methods 2010 4.42
10 In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol 2008 3.79
11 Biological basis for restriction of microRNA targets to the 3' untranslated region in mammalian mRNAs. Nat Struct Mol Biol 2009 3.70
12 Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol 2004 3.65
13 Human tRNA-derived small RNAs in the global regulation of RNA silencing. RNA 2010 3.40
14 Sarcoma derived from cultured mesenchymal stem cells. Stem Cells 2006 3.40
15 Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol 2005 3.28
16 Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol 2003 3.15
17 High-resolution genome-wide mapping of transposon integration in mammals. Mol Cell Biol 2005 3.10
18 Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 2003 2.95
19 The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J Virol 2006 2.94
20 Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014 2.92
21 Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration. J Clin Invest 2011 2.83
22 AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet 2003 2.81
23 Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 2003 2.74
24 Distinct pathways of genomic progression to benign and malignant tumors of the liver. Proc Natl Acad Sci U S A 2007 2.56
25 Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol 2002 2.54
26 Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther 2003 2.49
27 In vivo activity of nuclease-resistant siRNAs. RNA 2004 2.33
28 Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat Biotechnol 2002 2.31
29 Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. J Virol 2005 2.09
30 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 2.06
31 Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Mol Ther 2008 2.01
32 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 1.97
33 Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest 2010 1.91
34 The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo. Cell 2012 1.84
35 Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circ Res 2012 1.84
36 Site-directed transposon integration in human cells. Nucleic Acids Res 2007 1.74
37 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 1.71
38 Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver. Mol Ther 2007 1.51
39 Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice. Mol Ther 2002 1.51
40 Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo. Mol Ther 2007 1.51
41 Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Mol Ther 2008 1.49
42 Novel minicircle vector for gene therapy in murine myocardial infarction. Circulation 2009 1.45
43 Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol Ther 2003 1.42
44 Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology 2010 1.41
45 Combinatorial RNAi: a winning strategy for the race against evolving targets? Mol Ther 2007 1.40
46 In vivo correction of murine tyrosinemia type I by DNA-mediated transposition. Mol Ther 2002 1.39
47 Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells. Mol Cell Biol 2003 1.37
48 Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J Virol 2006 1.36
49 Immunogenicity and immunomodulatory properties of umbilical cord lining mesenchymal stem cells. Cell Transplant 2010 1.36
50 The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. J Virol 2007 1.35
51 Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? J Clin Invest 2007 1.34
52 Capped small RNAs and MOV10 in human hepatitis delta virus replication. Nat Struct Mol Biol 2008 1.34
53 In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus. J Clin Invest 2003 1.33
54 Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia. J Biol Chem 2008 1.32
55 The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver. Mol Ther 2008 1.31
56 Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Nat Protoc 2010 1.30
57 Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes. Proc Natl Acad Sci U S A 2011 1.28
58 FATP2 is a hepatic fatty acid transporter and peroxisomal very long-chain acyl-CoA synthetase. Am J Physiol Endocrinol Metab 2010 1.26
59 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 1.26
60 Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration. Hum Gene Ther 2006 1.25
61 Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther 2012 1.24
62 Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo. Hum Gene Ther 2004 1.22
63 A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8. Mol Ther 2007 1.19
64 A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood 2002 1.18
65 How do miRNAs mediate translational repression? Silence 2010 1.17
66 Postintegrative gene silencing within the Sleeping Beauty transposition system. Mol Cell Biol 2007 1.15
67 Adeno-associated virus vectors for short hairpin RNA expression. Methods Enzymol 2005 1.14
68 Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases. Hepatology 2005 1.14
69 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 1.14
70 A prenylation inhibitor prevents production of infectious hepatitis delta virus particles. J Virol 2002 1.13
71 RNAi and gene therapy: a mutual attraction. Hematology Am Soc Hematol Educ Program 2007 1.10
72 Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice. Mol Ther 2009 1.09
73 Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice. Mol Ther 2003 1.08
74 Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. Mol Ther 2010 1.08
75 Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells. Blood 2006 1.08
76 Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Mol Ther 2012 1.08
77 Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo. J Virol 2003 1.06
78 A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro. Mol Ther 2013 1.05
79 Radioprotection in vitro and in vivo by minicircle plasmid carrying the human manganese superoxide dismutase transgene. Hum Gene Ther 2008 1.04
80 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 1.04
81 Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency. Mol Ther 2007 1.02
82 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 1.02
83 Looking into the safety of AAV vectors. Nature 2003 1.02
84 Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo. Hum Gene Ther 2005 1.01
85 A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood 2003 1.00
86 Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B. Mol Ther 2010 1.00
87 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 0.98
88 A rapid protocol for construction and production of high-capacity adenoviral vectors. Nat Protoc 2009 0.97
89 Slicing-independent RISC activation requires the argonaute PAZ domain. Curr Biol 2012 0.96
90 Combined proteomic-RNAi screen for host factors involved in human hepatitis delta virus replication. RNA 2009 0.95
91 Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Hum Gene Ther Clin Dev 2015 0.95
92 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 0.95
93 Expression determinants of mammalian argonaute proteins in mediating gene silencing. Nucleic Acids Res 2011 0.95
94 The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo. Mol Ther 2003 0.94
95 AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Mol Ther 2012 0.93
96 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 0.93
97 Optimization of cis-acting elements for gene expression from nonviral vectors in vivo. Hum Gene Ther 2003 0.92
98 Advancing molecular therapies through in vivo bioluminescent imaging. Mol Imaging 2003 0.92
99 An in vitro-identified high-affinity nucleosome-positioning signal is capable of transiently positioning a nucleosome in vivo. Epigenetics Chromatin 2010 0.91
100 A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice. Hepatology 2003 0.91
101 Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor. J Cardiovasc Pharmacol 2009 0.91
102 Real-time in vivo imaging of stem cells following transgenesis by transposition. Mol Ther 2005 0.89
103 Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors. Hum Gene Ther 2007 0.89
104 miR-122 continues to blaze the trail for microRNA therapeutics. Mol Ther 2010 0.88
105 Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Mol Ther 2007 0.88
106 Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo. Hum Gene Ther 2002 0.88
107 MicroRNAs in Cancer: the 22nd Hiroshima Cancer Seminar/the 4th Japanese Association for RNA Interference Joint International Symposium, 30 August 2012, Grand Prince Hotel Hiroshima. Jpn J Clin Oncol 2013 0.88
108 Wandering eye for RNAi. Nat Med 2008 0.85
109 Donor-derived, liver-specific protein expression after bone marrow transplantation. Transplantation 2004 0.84
110 Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium. Hum Gene Ther 2007 0.82
111 Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice. Biotechnol Prog 2003 0.81
112 The expanding repertoire of circular RNAs. Mol Ther 2013 0.81
113 rAAV-mediated tumorigenesis: still unresolved after an AAV assault. Mol Ther 2012 0.81
114 Stability and repeat regeneration potential of the engineered liver tissues under the kidney capsule in mice. Cell Transplant 2005 0.80
115 Hepatic parenchymal replacement in mice by transplanted allogeneic hepatocytes is facilitated by bone marrow transplantation and mediated by CD4 cells. Hepatology 2008 0.79
116 Efficient inhibition of in-stent restenosis by controlled stent-based inhibition of elastase: a pilot study. J Vasc Interv Radiol 2004 0.78
117 The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA. Nucleic Acids Res 2013 0.78
118 Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver. Hum Gene Ther 2005 0.78
119 Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs. Hum Gene Ther Clin Dev 2014 0.77
120 System for simultaneous tissue-specific and disease-specific regulation of therapeutic gene expression. Hum Gene Ther 2003 0.76
121 A story of mice and men. Gene Ther 2002 0.76
122 Viral Vectors Take On HIV Infection. N Engl J Med 2015 0.75
123 Characterization of vector-based delivery of neurogenin-3 in murine diabetes. Hum Gene Ther 2014 0.75
124 Good news on the clinical gene transfer front. Hum Gene Ther 2008 0.75
125 A Tribute to George Stamatoyannopoulos. Hum Gene Ther 2016 0.75
126 Prime time for small RNA-based therapeutics. Hum Gene Ther 2008 0.75