Published in Neurogenesis (Austin) on December 03, 2015
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
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
Genome editing. The new frontier of genome engineering with CRISPR-Cas9. Science (2014) 10.43
ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES. Science (1965) 9.77
Optogenetics in neural systems. Neuron (2011) 7.56
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
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet (2007) 7.01
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 6.71
Astroglia induce neurogenesis from adult neural stem cells. Nature (2002) 6.41
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther (2008) 5.83
Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci U S A (2000) 4.39
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet (2014) 4.24
AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol (2011) 4.23
Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol (2008) 4.16
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol (2006) 4.12
Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol (2010) 3.84
In vivo clonal analysis reveals self-renewing and multipotent adult neural stem cell characteristics. Cell (2011) 3.47
Dynamic contribution of nestin-expressing stem cells to adult neurogenesis. J Neurosci (2007) 3.37
Neuroscience. The brain activity map. Science (2013) 3.36
In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9. Nat Biotechnol (2014) 2.51
Characterization of the DNA of a defective human parvovirus isolated from a genital site. Virology (1984) 2.49
Small deoxyribonucleic acid-containing viruses (picodnavirus group). Nature (1966) 2.45
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
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology (2009) 2.37
AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method. Mol Ther (2007) 2.17
Isolation of adenovirus-associated viruses from man. Proc Natl Acad Sci U S A (1967) 2.10
Beta-catenin signaling is required for neural differentiation of embryonic stem cells. Development (2004) 1.92
Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein. Virology (2004) 1.88
In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med (2013) 1.86
Immune responses to AAV in a phase I study for Canavan disease. J Gene Med (2006) 1.74
DNA shuffling of adeno-associated virus yields functionally diverse viral progeny. Mol Ther (2008) 1.70
A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol Ther (2010) 1.68
Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther (2009) 1.61
The presence of FGF2 signaling determines whether beta-catenin exerts effects on proliferation or neuronal differentiation of neural stem cells. Dev Biol (2004) 1.59
Molecular evolution of adeno-associated virus for enhanced glial gene delivery. Mol Ther (2009) 1.57
Structure of adeno-associated virus type 4. J Virol (2005) 1.47
Ablation of Fmrp in adult neural stem cells disrupts hippocampus-dependent learning. Nat Med (2011) 1.47
Directed evolution of adeno-associated virus to an infectious respiratory virus. Proc Natl Acad Sci U S A (2009) 1.42
A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Müller cells. PLoS One (2009) 1.40
Enhanced gene transfer efficiency in the murine striatum and an orthotopic glioblastoma tumor model, using AAV-7- and AAV-8-pseudotyped vectors. Hum Gene Ther (2006) 1.34
Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors. Gene Ther (2004) 1.32
Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. Mol Ther (2010) 1.31
MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther (2010) 1.30
Directed evolution of adeno-associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells. Mol Ther (2011) 1.28
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver. Gene Ther (2010) 1.21
Astrocytes regulate adult hippocampal neurogenesis through ephrin-B signaling. Nat Neurosci (2012) 1.19
microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors. Gene Ther (2010) 1.16
Long-distance axonal regeneration induced by CNTF gene transfer is impaired by axonal misguidance in the injured adult optic nerve. Neurobiol Dis (2012) 1.10
AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa. Mol Ther (2011) 1.10
Brain-derived neurotrophic factor stimulates proliferation and differentiation of neural stem cells, possibly by triggering the Wnt/β-catenin signaling pathway. J Neurosci Res (2012) 1.00
Overview: engineering transgenic constructs and mice. Curr Protoc Cell Biol (2009) 0.98
An evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cells. Mol Ther (2011) 0.95
CRALBP supports the mammalian retinal visual cycle and cone vision. J Clin Invest (2015) 0.90
Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery. J Gene Med (2013) 0.84
Enhanced selective gene delivery to neural stem cells in vivo by an adeno-associated viral variant. Development (2015) 0.76