Published in JAMA Ophthalmol on April 01, 2014
Biology and therapy of inherited retinal degenerative disease: insights from mouse models. Dis Model Mech (2015) 1.05
Manufacturing of recombinant adeno-associated viral vectors for clinical trials. Mol Ther Methods Clin Dev (2016) 1.05
Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results. Ophthalmology (2015) 0.97
Vector platforms for gene therapy of inherited retinopathies. Prog Retin Eye Res (2014) 0.94
Efficacy and Safety of rAAV2-ND4 Treatment for Leber's Hereditary Optic Neuropathy. Sci Rep (2016) 0.91
LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile. Invest Ophthalmol Vis Sci (2014) 0.90
Mechanisms of mesenchymal stem/stromal cell function. Stem Cell Res Ther (2016) 0.88
Gene therapy of inherited retinal degenerations: prospects and challenges. Hum Gene Ther (2015) 0.87
Clinical development of gene therapy: results and lessons from recent successes. Mol Ther Methods Clin Dev (2016) 0.85
Mitochondrial cytochrome c oxidase deficiency. Clin Sci (Lond) (2016) 0.84
Nuclear expression of mitochondrial ND4 leads to the protein assembling in complex I and prevents optic atrophy and visual loss. Mol Ther Methods Clin Dev (2015) 0.80
Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice. Mol Ther Methods Clin Dev (2016) 0.80
Correction of Monogenic and Common Retinal Disorders with Gene Therapy. Genes (Basel) (2017) 0.79
Cell-permeable protein therapy for complex I dysfunction. J Bioenerg Biomembr (2014) 0.77
Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus. Hum Gene Ther (2014) 0.77
Stable nuclear expression of ATP8 and ATP6 genes rescues a mtDNA Complex V null mutant. Nucleic Acids Res (2016) 0.75
Is there treatment for Leber hereditary optic neuropathy? Curr Opin Ophthalmol (2015) 0.75
Significant changes in endogenous retinal gene expression assessed 1 year after a single intraocular injection of AAV-CNTF or AAV-BDNF. Mol Ther Methods Clin Dev (2016) 0.75
Chapter 3 - Restoring Vision to the Blind: Gene Therapy for Vision Loss. Transl Vis Sci Technol (2014) 0.75
A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors. 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
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
Protein import into mitochondria. Annu Rev Biochem (1997) 6.82
CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med (2007) 5.24
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
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood (2002) 4.60
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther (2001) 4.35
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A (2008) 3.84
Detergent binding explains anomalous SDS-PAGE migration of membrane proteins. Proc Natl Acad Sci U S A (2009) 3.74
Mitochondrial optic neuropathies - disease mechanisms and therapeutic strategies. Prog Retin Eye Res (2010) 3.58
A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain (2011) 3.48
Self-complementary AAV vectors; advances and applications. Mol Ther (2008) 3.10
In vivo three-dimensional high-resolution imaging of rodent retina with spectral-domain optical coherence tomography. Invest Ophthalmol Vis Sci (2007) 2.89
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
Rescue of a deficiency in ATP synthesis by transfer of MTATP6, a mitochondrial DNA-encoded gene, to the nucleus. Nat Genet (2002) 2.20
Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy. Ann Neurol (2002) 2.13
Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain. J Virol (2004) 2.12
Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression. Virology (2008) 1.96
Treatment for mitochondrial disorders. Cochrane Database Syst Rev (2012) 1.94
Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model. Proc Natl Acad Sci U S A (2012) 1.94
Import of proteins into mitochondria. Annu Rev Genet (1991) 1.84
High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol Ther (2010) 1.78
Immunity to adeno-associated virus vectors in animals and humans: a continued challenge. Gene Ther (2008) 1.73
Production and purification of recombinant adeno-associated virus. Methods Enzymol (2000) 1.66
Electrophoresis techniques to investigate defects in oxidative phosphorylation. Methods (2008) 1.63
Resolution of the membrane domain of bovine complex I into subcomplexes: implications for the structural organization of the enzyme. Biochemistry (2000) 1.61
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
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest (2009) 1.54
Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders. Mol Ther (2006) 1.54
Heteroplasmy in Leber's hereditary optic neuropathy. Arch Ophthalmol (1993) 1.51
The pattern electroretinogram as a tool to monitor progressive retinal ganglion cell dysfunction in the DBA/2J mouse model of glaucoma. Invest Ophthalmol Vis Sci (2007) 1.35
The mouse pattern electroretinogram. Doc Ophthalmol (2007) 1.34
The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse. Invest Ophthalmol Vis Sci (2007) 1.26
Preclinical safety evaluation of AAV2-sFLT01- a gene therapy for age-related macular degeneration. Mol Ther (2010) 1.22
Gene therapy in cystic fibrosis. Chest (2001) 1.19
Assembly of functional proton-translocating ATPase complex in yeast mitochondria with cytoplasmically synthesized subunit 8, a polypeptide normally encoded within the organelle. Proc Natl Acad Sci U S A (1988) 1.17
Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system. Invest Ophthalmol Vis Sci (2009) 1.14
Effect of EPI-743 on the clinical course of the mitochondrial disease Leber hereditary optic neuropathy. Arch Neurol (2012) 1.12
Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1. Arch Ophthalmol (2010) 1.02
Rapid purification and mass spectrometric characterization of mitochondrial NADH dehydrogenase (Complex I) from rodent brain and a dopaminergic neuronal cell line. Mol Cell Proteomics (2004) 1.00
Leber's Hereditary Optic Neuropathy-Gene Therapy: From Benchtop to Bedside. J Ophthalmol (2010) 0.96
The optimized allotopic expression of ND1 or ND4 genes restores respiratory chain complex I activity in fibroblasts harboring mutations in these genes. Biochim Biophys Acta (2008) 0.94
Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus. Arch Ophthalmol (2010) 0.93
Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina. Mol Vis (2009) 0.85
Allotopic expression of mitochondrial ATP synthase genes in nucleus of Saccharomyces cerevisiae. Methods Enzymol (1996) 0.80
Quality of Life in the Tube Versus Trabeculectomy Study. Am J Ophthalmol (2017) 0.75
Tube Fenestration in the Tube Versus Trabeculectomy Study. Ophthalmology (2016) 0.75