Search Results - "Koilkonda, Rajeshwari"
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Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results
Published in Ophthalmology (Rochester, Minn.) (01-11-2017)“…To determine the effects of AAV2(Y444,500,730F)-P1ND4v2 in patients with Leber hereditary optic neuropathy (LHON). Prospective open-label, unilateral…”
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Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results
Published in Ophthalmology (Rochester, Minn.) (01-03-2016)“…Leber hereditary optic neuropathy (LHON) is a disorder characterized by severe and rapidly progressive visual loss when caused by a mutation in the…”
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Gene Therapy with Single-Subunit Yeast NADH-Ubiquinone Oxidoreductase (NDI1) Improves the Visual Function in Experimental Autoimmune Encephalomyelitis (EAE) Mice Model of Multiple Sclerosis (MS)
Published in Molecular neurobiology (01-04-2020)“…Mitochondrial dysfunction mediated loss of respiration, oxidative stress, and loss of cellular homeostasis contributes to the neuronal and axonal degenerations…”
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Gene Therapy for Leber Hereditary Optic Neuropathy
Published in Ophthalmology (Rochester, Minn.) (01-11-2017)“…Purpose To determine the effects of AAV2(Y444,500,730F)-P1ND4v2 in patients with Leber hereditary optic neuropathy (LHON). Design Prospective open-label,…”
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Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model
Published in Proceedings of the National Academy of Sciences - PNAS (15-05-2012)“…To introduce DNA into mitochondria efficiently, we fused adenoassociated virus capsid VP2 with a mitochondrial targeting sequence to carry the mitochondrial…”
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Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial
Published in JAMA ophthalmology (01-04-2014)“…IMPORTANCE We developed a novel strategy for treatment of Leber hereditary optic neuropathy (LHON) caused by a mutation in the nicotinamide adenine…”
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Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice
Published in Proceedings of the National Academy of Sciences - PNAS (20-10-2015)“…Considerable evidence supports mutations in mitochondrial genes as the cause of maternally inherited diseases affecting tissues that rely primarily on…”
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Noninvasive assessments of optic nerve neurodegeneration in transgenic mice with isolated optic neuritis
Published in Investigative ophthalmology & visual science (01-07-2013)“…To determine if phosphorylated neurofilament heavy chain (pNF-H) released into the bloodstream and the pattern ERG are noninvasive indicators of…”
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Efficiency and Safety of AAV-Mediated Gene Delivery of the Human ND4 Complex I Subunit in the Mouse Visual System
Published in Investigative ophthalmology & visual science (01-09-2009)“…To evaluate the efficiency and safety of AAV-mediated gene delivery of a normal human ND4 complex I subunit in the mouse visual system. A nuclear encoded human…”
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Leber's Hereditary Optic Neuropathy-Gene Therapy : From Benchtop to Bedside
Published in Journal of ophthalmology (01-01-2011)“…Leber's hereditary optic neuropathy (LHON) is a maternally transmitted disorder caused by point mutations in mitochondrial DNA (mtDNA). Most cases are due to…”
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Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice
Published in Molecular vision (20-06-2012)“…Although mutated G11778A NADH ubiquinone oxidoreductase subunit 4 (ND4) mitochondrial DNA (mtDNA) is firmly linked to the blindness of Leber hereditary optic…”
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Gene Therapy for Leber Hereditary Optic Neuropathy
Published in Ophthalmology (Rochester, Minn.) (01-03-2016)“…Purpose Leber hereditary optic neuropathy (LHON) is a disorder characterized by severe and rapidly progressive visual loss when caused by a mutation in the…”
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Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina
Published in Molecular vision (16-12-2009)“…To evaluate the efficiency of self-complementary adeno-associated virus (scAAV)-mediated gene expression of green fluorescent protein (GFP) or the allotopic…”
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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
Published in Archives of ophthalmology (1960) (01-07-2010)“…To demonstrate the high efficiency and rapidity of allotopic expression of a normal human ND4 subunit of complex I in the vertebrate retina using a…”
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Targeted Krüppel-Like Factor 4 Gene Knock-Out in Retinal Ganglion Cells Improves Visual Function in Multiple Sclerosis Mouse Model
Published in eNeuro (01-03-2020)“…Axonal demyelination injury and neuronal degeneration are the primary causes of visual disability in multiple sclerosis (MS)-linked optic neuritis patients…”
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Low- and Medium-Dose Gene Therapy in Patients with Prior Unilateral LHON Does Not Prevent Further Visual Loss in the Treated Eye (S16.006)
Published in Neurology (10-04-2018)“…Abstract only…”
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Gene Therapy for LHON : Low and Medium Dose Results (S26.004)
Published in Neurology (18-04-2017)“…Abstract only…”
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LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile
Published in Investigative ophthalmology & visual science (01-12-2014)“…To demonstrate safety and efficacy of allotopic human ND4 for treatment of a Leber's hereditary optic neuropathy (LHON) mouse model harboring the G11778A…”
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