PubRank

R J Samulski

Author PubWeight™ 182.44‹?›

Top papers

Rank Title Journal Year PubWeight™‹?›
1 Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998 8.45
2 Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther 1999 7.96
3 Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998 7.76
4 Site-specific integration by adeno-associated virus. Proc Natl Acad Sci U S A 1990 7.06
5 Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996 6.43
6 Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 1996 6.22
7 Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci U S A 1982 5.73
8 Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 1994 5.49
9 Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV. Cell 1983 5.27
10 AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nat Med 1999 5.26
11 Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 2003 4.92
12 A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J Virol 1987 4.57
13 Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 2001 4.35
14 Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors. J Virol 2000 3.84
15 Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther 2000 3.32
16 In vitro resolution of covalently joined AAV chromosome ends. Cell 1990 3.24
17 Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res 1996 3.03
18 Features of the adeno-associated virus origin involved in substrate recognition by the viral Rep protein. J Virol 1993 2.69
19 Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J Virol 1997 2.32
20 Role for highly regulated rep gene expression in adeno-associated virus vector production. J Virol 1997 2.19
21 Identification of a repeated sequence element required for efficient encapsidation of the adenovirus type 5 chromosome. J Virol 1987 2.09
22 Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum Gene Ther 1998 2.06
23 Colocalization of adeno-associated virus Rep and capsid proteins in the nuclei of infected cells. J Virol 1992 1.86
24 Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector. Proc Natl Acad Sci U S A 1992 1.82
25 Insertional mutagenesis of AAV2 capsid and the production of recombinant virus. Virology 1999 1.76
26 Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood 1994 1.75
27 Immune responses to AAV in a phase I study for Canavan disease. J Gene Med 2006 1.74
28 Gene transfer by adeno-associated virus vectors into the central nervous system. Exp Neurol 1997 1.73
29 The role of Kupffer cell oxidant production in early ethanol-induced liver disease. Free Radic Biol Med 2001 1.69
30 Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors. Mol Ther 2001 1.65
31 Rapid purification of covalently closed circular DNAs of bacterial plasmids and animal tumor viruses. Anal Biochem 1980 1.60
32 A novel 165-base-pair terminal repeat sequence is the sole cis requirement for the adeno-associated virus life cycle. J Virol 1997 1.57
33 Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther 1998 1.52
34 Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody. Nat Biotechnol 1999 1.51
35 Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther 2011 1.47
36 AAV vectors: is clinical success on the horizon? Gene Ther 2000 1.47
37 Adeno-associated virus vectors for gene therapy: more pros than cons? Mol Med Today 2000 1.45
38 Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J Clin Invest 1994 1.39
39 Roles of adeno-associated virus Rep protein and human chromosome 19 in site-specific recombination. J Virol 2000 1.37
40 Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element. J Virol 2000 1.32
41 Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells. Proc Natl Acad Sci U S A 1994 1.28
42 Adeno-associated viral vectors as gene delivery vehicles. Int J Mol Med 2000 1.25
43 Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. Gene Ther 2011 1.24
44 New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. Nat Med 1997 1.21
45 Adeno-associated virus (AAV) site-specific recombination does not require a Rep-dependent origin of replication within the AAV terminal repeat. Proc Natl Acad Sci U S A 2001 1.21
46 Building a better vector: the manipulation of AAV virions. Virology 2000 1.19
47 Factors influencing adeno-associated virus-mediated gene transfer to human cystic fibrosis airway epithelial cells: comparison with adenovirus vectors. J Virol 1998 1.17
48 Construction of a recombinant human parvovirus B19: adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus. Proc Natl Acad Sci U S A 1989 1.17
49 Long-term gene transfer in porcine myocardium after coronary infusion of an adeno-associated virus vector. Ann Thorac Surg 1996 1.16
50 In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther 1998 1.16
51 Gene therapy for human hemoglobinopathies. Proc Soc Exp Biol Med 1993 1.15
52 The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers. Hum Gene Ther 2000 1.14
53 Single-copy transduction and expression of human gamma-globin in K562 erythroleukemia cells using recombinant adeno-associated virus vectors: the effect of mutations in NF-E2 and GATA-1 binding motifs within the hypersensitivity site 2 enhancer. Blood 1993 1.12
54 Inducible long-term gene expression in brain with adeno-associated virus gene transfer. Gene Ther 1998 1.09
55 Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs. Gene Ther 2012 1.09
56 Gene therapy: targeting the myocardium. Heart 2008 1.06
57 Adeno-associated virus-mediated expression and constitutive secretion of NPY or NPY13-36 suppresses seizure activity in vivo. Gene Ther 2007 1.05
58 Delivery of MDR1 small interfering RNA by self-complementary recombinant adeno-associated virus vector. Mol Ther 2005 1.05
59 NIDDK Workshop on AAV Vectors: Gene Transfer into Quiescent Cells. Hum Gene Ther 1996 0.98
60 Efficient expression of protein coding genes from the murine U1 small nuclear RNA promoters. Proc Natl Acad Sci U S A 1996 0.98
61 Kinetics of recombinant adeno-associated virus-mediated gene transfer. J Virol 2000 0.98
62 Gene transfer in human lymphocytes using a vector based on adeno-associated virus. J Immunother (1991) 1992 0.98
63 Delivery of the Cu/Zn-superoxide dismutase gene with adenovirus reduces early alcohol-induced liver injury in rats. Gastroenterology 2001 0.98
64 Adeno-associated virus-mediated gene transfer of the heart/muscle adenine nucleotide translocator (ANT) in mouse. Gene Ther 2005 0.97
65 Ex vivo serotype-specific transduction of equine joint tissue by self-complementary adeno-associated viral vectors. Hum Gene Ther 2009 0.97
66 Fluorescent viral vectors: a new technique for the pharmacological analysis of gene therapy. Nat Med 1998 0.97
67 Adeno-associated virus (AAV) vector antisense gene transfer in vivo decreases GABA(A) alpha1 containing receptors and increases inferior collicular seizure sensitivity. Brain Res 1997 0.96
68 Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair. Gene Ther 2010 0.95
69 Heart failure: a silver bullet to treat heart failure. Gene Ther 2006 0.95
70 Embryonic substantia nigra grafts in the mesencephalon send neurites to the host striatum in non-human primate after overexpression of GDNF. J Comp Neurol 2009 0.92
71 Viral receptors and vector purification: new approaches for generating clinical-grade reagents. Nat Med 1999 0.91
72 Preclinical safety evaluation of subretinal AAV2.sFlt-1 in non-human primates. Gene Ther 2011 0.91
73 Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals. Gene Ther 2008 0.90
74 The influence of epileptic neuropathology and prior peripheral immunity on CNS transduction by rAAV2 and rAAV5. Gene Ther 2011 0.90
75 AAV as a viral vector for human gene therapy. Generation of recombinant virus. Mol Biotechnol 1995 0.89
76 AAV2-mediated gene transfer of GDNF to the striatum of MPTP monkeys enhances the survival and outgrowth of co-implanted fetal dopamine neurons. Exp Neurol 2008 0.88
77 Cu/Zn-superoxide dismutase gene attenuates ischemia-reperfusion injury in the rat kidney. J Am Soc Nephrol 2001 0.87
78 A small regulatory element from chromosome 19 enhances liver-specific gene expression. Gene Ther 2008 0.87
79 Comparison of the effect of adenoviral delivery of three superoxide dismutase genes against hepatic ischemia-reperfusion injury. Hum Gene Ther 2001 0.86
80 Charge-to-alanine mutagenesis of the adeno-associated virus type 2 Rep78/68 proteins yields temperature-sensitive and magnesium-dependent variants. J Virol 1999 0.86
81 Site-specific targeting of DNA plasmids to chromosome 19 using AAV cis and trans sequences. Methods Mol Biol 2000 0.85
82 Efficient transduction of vascular smooth muscle cells with a translational AAV2.5 vector: a new perspective for in-stent restenosis gene therapy. Gene Ther 2013 0.83
83 Three-dimensional multipotent progenitor cell aggregates for expansion, osteogenic differentiation and 'in vivo' tracing with AAV vector serotype 6. Gene Ther 2012 0.83
84 Delivery of Cu/Zn-superoxide dismutase genes with a viral vector minimizes liver injury and improves survival after liver transplantation in the rat. Transplantation 2000 0.80
85 Chronic ethanol increases adeno-associated viral transgene expression in rat liver via oxidant and NFkappaB-dependent mechanisms. Hepatology 2000 0.80
86 Expanding the AAV package. Nat Biotechnol 2000 0.80
87 The genetics of adeno-associated virus. Adv Exp Med Biol 1984 0.79
88 Methods for adeno-associated virus-mediated gene transfer into muscle. Methods Mol Biol 2001 0.79
89 Delivering multiple gene products in the brain from a single adeno-associated virus vector. Gene Ther 2009 0.78
90 Efficient gene transfer into primary and immortalized human fetal glial cells using adeno-associated virus vectors: establishment of a glial cell line with a functional CD4 receptor. J Neurovirol 1997 0.76
91 Gene therapy for cardiomyocytes, a heart beat away. Gene Ther 2009 0.76
92 Vector biophysics: crystal-clear view. Gene Ther 2002 0.75
93 Methods for the construction and propagation of recombinant adeno-associated virus vectors. Methods Mol Med 1997 0.75
94 Expression of the human glucocerebrosidase and arylsulfatase A genes in murine and patient primary fibroblasts transduced by an adeno-associated virus vector. Gene Ther 1994 0.75
95 Adeno-associated virus vectors for gene transfer into erythroid cells. Curr Top Microbiol Immunol 1996 0.75