Published in Mol Ther on November 01, 2013
State-of-the-art human gene therapy: part I. Gene delivery technologies. Discov Med (2014) 0.86
CRISPR/Cas9-Mediated Genome Editing of Herpesviruses Limits Productive and Latent Infections. PLoS Pathog (2016) 0.80
CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector. Mol Ther Nucleic Acids (2016) 0.79
Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction. Proc Natl Acad Sci U S A (2015) 0.77
Adenovirus-Mediated Gene Delivery: Potential Applications for Gene and Cell-Based Therapies in the New Era of Personalized Medicine. Genes Dis (2017) 0.75
Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 17.21
Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med (2008) 15.30
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A (2002) 11.11
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med (2011) 11.01
ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES. Science (1965) 9.77
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther (2008) 8.83
Nucleotide sequence and organization of the adeno-associated virus 2 genome. J Virol (1983) 7.52
Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics. Proc Natl Acad Sci U S A (1966) 7.35
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A (2008) 7.16
Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol (2004) 7.12
Site-specific integration by adeno-associated virus. Proc Natl Acad Sci U S A (1990) 7.06
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 6.71
Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol (1989) 6.69
Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J (1991) 6.13
The AAV origin binding protein Rep68 is an ATP-dependent site-specific endonuclease with DNA helicase activity. Cell (1990) 5.98
Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J Virol (2002) 5.82
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet (2000) 5.80
Evidence for a single-stranded adenovirus-associated virus genome: formation of a DNA density hybrid on release of viral DNA. Proc Natl Acad Sci U S A (1969) 5.78
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
Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet (1994) 5.49
Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV. Cell (1983) 5.27
Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells. J Virol (1980) 5.09
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet (1997) 4.72
Gene therapy vectors based on adeno-associated virus type 1. J Virol (1999) 4.63
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood (2002) 4.60
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
Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J (1992) 4.36
Cloning of infectious adeno-associated virus genomes in bacterial plasmids. Gene (1983) 4.15
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther (2009) 3.93
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol (2011) 3.79
Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci U S A (1993) 3.59
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A (2009) 3.58
Factors that bind to adeno-associated virus terminal repeats. J Virol (1989) 3.56
Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter. Genomics (1991) 3.47
Serologic evidence for human infection with adenovirus-associated viruses. J Natl Cancer Inst (1968) 3.44
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med (1999) 3.39
Negative and positive regulation in trans of gene expression from adeno-associated virus vectors in mammalian cells by a viral rep gene product. Mol Cell Biol (1986) 3.34
Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther (2000) 3.32
Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proc Natl Acad Sci U S A (2003) 3.31
In vitro resolution of covalently joined AAV chromosome ends. Cell (1990) 3.24
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther (2009) 3.07
Defective regulation of outwardly rectifying Cl- channels by protein kinase A corrected by insertion of CFTR. Nature (1992) 3.06
Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J Biol Chem (1993) 3.02
AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med (2012) 2.87
Partial purification of adeno-associated virus Rep78, Rep52, and Rep40 and their biochemical characterization. J Virol (1992) 2.84
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. Hum Gene Ther (2011) 2.75
Features of the adeno-associated virus origin involved in substrate recognition by the viral Rep protein. J Virol (1993) 2.69
Evidence for covalent attachment of the adeno-associated virus (AAV) rep protein to the ends of the AAV genome. J Virol (1990) 2.62
Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med (2009) 2.57
Expression from the adeno-associated virus p5 and p19 promoters is negatively regulated in trans by the rep protein. J Virol (1989) 2.53
Organization of adeno-associated virus DNA in latently infected Detroit 6 cells. Virology (1989) 2.43
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther (2008) 2.42
In vivo model of adeno-associated virus vector persistence and rescue. J Virol (1996) 2.41
Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Mol Ther (2012) 2.40
A human parvovirus, adeno-associated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase. Mol Cell Biol (1984) 2.36
Adeno-associated viruses. Adv Virus Res (1979) 2.35
Identification of a DNA-binding domain in the amino terminus of adeno-associated virus Rep proteins. J Virol (1993) 2.22
Isolation of adenovirus-associated viruses from man. Proc Natl Acad Sci U S A (1967) 2.10
Alternate mRNA splicing is required for synthesis of adeno-associated virus VP1 capsid protein. J Virol (1988) 2.03
Epidemiology of adenovirus-associated virus infection in a nursery population. Am J Epidemiol (1968) 1.96
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology (2013) 1.85
Gene expression from adeno-associated virus vectors in airway epithelial cells. Am J Respir Cell Mol Biol (1992) 1.83
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther (2006) 1.83
A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther (1996) 1.80
Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol (1994) 1.80
Regulation of adeno-associated virus gene expression in 293 cells: control of mRNA abundance and translation. J Virol (1988) 1.80
Immune responses to AAV in a phase I study for Canavan disease. J Gene Med (2006) 1.74
A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol Ther (2010) 1.68
Adeno-associated virus DNA replication. Cold Spring Harb Symp Quant Biol (1979) 1.65
Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain. Hum Gene Ther (2002) 1.60
A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease. Hum Gene Ther (2001) 1.58
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther (2004) 1.54
Lessons learned from the clinical development and market authorization of Glybera. Hum Gene Ther Clin Dev (2013) 1.51
Analysis of adeno-associated virus (AAV) wild-type and mutant Rep proteins for their abilities to negatively regulate AAV p5 and p19 mRNA levels. J Virol (1994) 1.48
Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope (1999) 1.47
Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther (1996) 1.45
Biochemical characterization of adeno-associated virus rep68 DNA helicase and ATPase activities. J Virol (1999) 1.45
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther (2002) 1.40
Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum Gene Ther (2003) 1.36
In vitro resolution of adeno-associated virus DNA hairpin termini by wild-type Rep protein is inhibited by a dominant-negative mutant of rep. J Virol (1992) 1.36
Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis. Hum Gene Ther (2004) 1.30
Inhibition of adenovirus oncogenicity in hamsters by adeno-associated virus DNA. J Natl Cancer Inst (1981) 1.30
Regulation of adeno-associated virus DNA replication. Biochim Biophys Acta (1988) 1.26
Parvovirus RNA transcripts containing sequences not present in mature mRNA: a method for isolation of putative mRNA precursor sequences. Proc Natl Acad Sci U S A (1979) 1.23
Gene therapy for aromatic L-amino acid decarboxylase deficiency. Sci Transl Med (2012) 1.22
Long-term follow-up after gene therapy for canavan disease. Sci Transl Med (2012) 1.22
Adeno-associated virus inhibits human papillomavirus type 16: a viral interaction implicated in cervical cancer. Cancer Res (1994) 1.19
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
Repeated delivery of adeno-associated virus vectors to the rabbit airway. J Virol (1999) 1.12
Interaction of wild-type and mutant adeno-associated virus (AAV) Rep proteins on AAV hairpin DNA. J Virol (1996) 1.06
A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. Hum Gene Ther (1998) 1.05
Effect of adeno-associated virus on transformation of NIH 3T3 cells by ras gene and on tumorigenicity of an NIH 3T3 transformed cell line. Cancer Res (1986) 0.98
Adeno-associated virus Rep78 inhibits oncogenic transformation of primary human keratinocytes by a human papillomavirus type 16-ras chimeric. Gynecol Oncol (1997) 0.92
Alipogene tiparvovec, an adeno-associated virus encoding the Ser(447)X variant of the human lipoprotein lipase gene for the treatment of patients with lipoprotein lipase deficiency. Curr Opin Mol Ther (2009) 0.92
Characterization of the adenoassociated virus Rep protein complex formed on the viral origin of DNA replication. Virology (2003) 0.91
Adeno-associated virus is associated with a lower risk of high-grade cervical neoplasia. Exp Mol Pathol (2001) 0.89
Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy. Hum Gene Ther (2005) 0.88
EFFICACY, POTENTIAL AND HAZARDS OF VACCINES. N Engl J Med (1964) 0.81
EXCRETION OF SV-40 VIRUS AFTER ORAL ADMINISTRATION OF CONTAMINATED POLIO VACCINE. Acta Microbiol Acad Sci Hung (1965) 0.78