Published in Sci Transl Med on March 03, 2010
AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med (2012) 2.87
Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies. J Clin Invest (2010) 2.13
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 2.09
AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models. PLoS One (2013) 1.62
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol Ther (2010) 1.35
Humoral Immune Response to AAV. Front Immunol (2013) 1.27
The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. Hum Gene Ther (2012) 1.16
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue. Gene Ther (2012) 1.06
Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery. PLoS One (2012) 0.96
Gene therapy for Leber congenital amaurosis: advances and future directions. Graefes Arch Clin Exp Ophthalmol (2012) 0.95
Direct gene transfer with compacted DNA nanoparticles in retinal pigment epithelial cells: expression, repeat delivery and lack of toxicity. Nanomedicine (Lond) (2012) 0.93
Clinical characteristics and current therapies for inherited retinal degenerations. Cold Spring Harb Perspect Med (2014) 0.92
Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates. Gene Ther (2014) 0.92
Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa. Hum Mol Genet (2013) 0.91
Immunology of AAV-Mediated Gene Transfer in the Eye. Front Immunol (2013) 0.90
Genomic DNA nanoparticles rescue rhodopsin-associated retinitis pigmentosa phenotype. FASEB J (2015) 0.87
AAV-mediated gene therapy in mouse models of recessive retinal degeneration. Curr Mol Med (2012) 0.84
Gene delivery to the retina: from mouse to man. Methods Enzymol (2012) 0.82
Dog models for blinding inherited retinal dystrophies. Hum Gene Ther Clin Dev (2015) 0.80
Evaluation of dose and safety of AAV7m8 and AAV8BP2 in the non-human primate retina. Hum Gene Ther (2016) 0.79
Reprogramming metabolism by targeting sirtuin 6 attenuates retinal degeneration. J Clin Invest (2016) 0.78
Adeno-Associated Virus-Mediated Gene Transfer to Renal Tubule Cells via a Retrograde Ureteral Approach. Nephron Extra (2011) 0.78
Gene Therapy: The View from NCATS. Hum Gene Ther (2016) 0.78
Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials. Front Neurosci (2017) 0.75
My career path for developing gene therapy for blinding diseases: the importance of mentors, collaborators, and opportunities. Hum Gene Ther (2014) 0.75
Protocols for Visually Guided Navigation Assessment of Efficacy of Retina-Directed Cell or Gene Therapy in Canines. Front Neurosci (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
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51
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
Gene therapy restores vision in a canine model of childhood blindness. Nat Genet (2001) 7.88
Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle. Nat Genet (1998) 7.57
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
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 6.71
Leber congenital amaurosis: genes, proteins and disease mechanisms. Prog Retin Eye Res (2008) 5.90
CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med (2007) 5.24
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther (2005) 4.74
Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle. Proc Natl Acad Sci U S A (2005) 4.67
Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther (1999) 4.65
RPE65 is the isomerohydrolase in the retinoid visual cycle. Proc Natl Acad Sci U S A (2005) 4.61
Rpe65 is the retinoid isomerase in bovine retinal pigment epithelium. Cell (2005) 4.51
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther (2009) 3.93
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther (2008) 3.28
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther (2009) 3.07
Congenital stationary night blindness in the dog: common mutation in the RPE65 gene indicates founder effect. Mol Vis (1998) 2.74
Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis. Invest Ophthalmol Vis Sci (2004) 2.72
Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients. Invest Ophthalmol Vis Sci (2007) 2.69
Early-onset severe rod-cone dystrophy in young children with RPE65 mutations. Invest Ophthalmol Vis Sci (2000) 2.67
Leber congenital amaurosis. Mol Genet Metab (1999) 2.52
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood (2007) 2.05
Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system. Invest Ophthalmol Vis Sci (2006) 2.01
Epidemiology of adenovirus-associated virus infection in a nursery population. Am J Epidemiol (1968) 1.96
The Briard dog: a new animal model of congenital stationary night blindness. Br J Ophthalmol (1989) 1.77
Diagnosis of ocular toxocariasis by establishing intraocular antibody production. Am J Ophthalmol (2007) 1.60
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential. Mol Vis (2008) 1.59
Molecular characterization of adeno-associated viruses infecting children. J Virol (2005) 1.46
Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice. J Gene Med (2009) 1.04
Additional transduction events after subretinal readministration of recombinant adeno-associated virus. Hum Gene Ther (2000) 1.02
Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction. Methods Enzymol (2000) 0.94
Specific antibody production in herpes keratitis: intraocular inflammation and corneal neovascularisation as predicting factors. Graefes Arch Clin Exp Ophthalmol (2005) 0.84
Using the NAFX to measure the effectiveness over time of gene therapy in canine LCA. Invest Ophthalmol Vis Sci (2009) 0.83
Intraocular antibody production in intraocular inflammation. Graefes Arch Clin Exp Ophthalmol (2000) 0.81
Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 17.21
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med (2011) 11.01
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 6.71
CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med (2007) 5.24
In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature (2011) 4.81
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther (2005) 4.74
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet (2011) 4.67
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood (2002) 4.60
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success. Proc Natl Acad Sci U S A (2005) 4.04
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther (2009) 3.93
AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood (2009) 3.65
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest (2003) 3.52
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood (2002) 3.45
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther (2008) 3.28
Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes. Mol Ther (2007) 3.26
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther (2011) 3.10
Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med (2014) 2.92
AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med (2012) 2.87
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med (2006) 2.78
Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis. Invest Ophthalmol Vis Sci (2004) 2.72
Alpha interferon induces distinct translational control programs to suppress hepatitis C virus RNA replication. J Virol (2003) 2.69
Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. Mol Vis (2003) 2.68
New ISSCR guidelines underscore major principles for responsible translational stem cell research. Cell Stem Cell (2008) 2.61
In utero gene therapy rescues vision in a murine model of congenital blindness. Mol Ther (2004) 2.56
Assessing the potential for AAV vector genotoxicity in a murine model. Blood (2010) 2.48
Kindlin-2 is an essential component of intercalated discs and is required for vertebrate cardiac structure and function. Circ Res (2008) 2.48
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol Ther (2005) 2.48
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest (2008) 2.40
Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther (2006) 2.33
Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence. Hum Mol Genet (2004) 2.30
Impact of the lung allocation score on lung transplantation for pulmonary arterial hypertension. Am J Respir Crit Care Med (2009) 2.19
Mutations of CASK cause an X-linked brain malformation phenotype with microcephaly and hypoplasia of the brainstem and cerebellum. Nat Genet (2008) 2.16
Lateral connectivity and contextual interactions in macaque primary visual cortex. Neuron (2002) 2.16
Immune responses to AAV in clinical trials. Curr Gene Ther (2007) 2.13
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol Ther (2006) 2.13
Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies. J Clin Invest (2010) 2.13
Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol (2008) 2.10
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer. Blood (2012) 2.10
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 2.09
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood (2007) 2.05
Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood (2008) 2.01
Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system. Invest Ophthalmol Vis Sci (2006) 2.01
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy. Blood (2008) 1.98
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood (2013) 1.98
Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum Gene Ther (2002) 1.98
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology (2013) 1.85
Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer. Blood (2004) 1.84
Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood (2004) 1.83
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther (2006) 1.81
Novel AAV serotypes for improved ocular gene transfer. J Gene Med (2008) 1.80
Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood (2004) 1.78
Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. Hum Gene Ther (2011) 1.71
Intrapulmonary pharmacokinetics of linezolid. Antimicrob Agents Chemother (2002) 1.65
Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirus. Vision Res (2003) 1.63
Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Mol Ther (2008) 1.63
Identification of mouse AAV capsid-specific CD8+ T cell epitopes. Mol Ther (2005) 1.63
AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models. PLoS One (2013) 1.62
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. Blood (2003) 1.62