Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy
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Title
Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy
Authors
Keywords
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Journal
BRAIN
Volume -, Issue -, Pages -
Publisher
Oxford University Press (OUP)
Online
2022-11-10
DOI
10.1093/brain/awac421
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- (2021) Mark L. Moster et al. JOURNAL OF NEURO-OPHTHALMOLOGY
- Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison
- (2021) Nancy J. Newman et al. Frontiers in Neurology
- Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates
- (2021) David J. Calkins et al. Molecular Therapy-Methods & Clinical Development
- Factors associated with rapid improvement in visual acuity in patients with Leber's hereditary optic neuropathy after gene therapy
- (2020) Hong‐li Liu et al. ACTA OPHTHALMOLOGICA
- Visual Outcomes in Leber Hereditary Optic Neuropathy Patients With the m.11778G>A (MTND4) Mitochondrial DNA Mutation
- (2020) Nancy J. Newman et al. JOURNAL OF NEURO-OPHTHALMOLOGY
- Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy
- (2020) Patrick Yu-Wai-Man et al. Science Translational Medicine
- Safety of rAAV2/2- ND4 Gene Therapy for Leber Hereditary Optic Neuropathy
- (2018) Catherine Vignal et al. OPHTHALMOLOGY
- Visual rehabilitation using microperimetric acoustic biofeedback training in individuals with central scotoma
- (2018) Dhanashree Ratra et al. Clinical and Experimental Optometry
- International Consensus Statement on the Clinical and Therapeutic Management of Leber Hereditary Optic Neuropathy
- (2017) Valerio Carelli et al. JOURNAL OF NEURO-OPHTHALMOLOGY
- Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
- (2017) Stephen Russell et al. LANCET
- Gene Therapy for Leber Hereditary Optic Neuropathy
- (2017) John Guy et al. OPHTHALMOLOGY
- A neurodegenerative perspective on mitochondrial optic neuropathies
- (2016) Patrick Yu-Wai-Man et al. ACTA NEUROPATHOLOGICA
- Gene Therapy for Leber Hereditary Optic Neuropathy
- (2016) William J. Feuer et al. OPHTHALMOLOGY
- Long-term outcomes of gene therapy for the treatment of Leber's hereditary optic neuropathy
- (2016) Shuo Yang et al. EBioMedicine
- Efficacy and Safety of rAAV2-ND4 Treatment for Leber’s Hereditary Optic Neuropathy
- (2016) Xing Wan et al. Scientific Reports
- Macular nerve fibre and ganglion cell layer changes in acute Leber's hereditary optic neuropathy
- (2015) Nicole Balducci et al. BRITISH JOURNAL OF OPHTHALMOLOGY
- Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis
- (2015) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
- Nuclear expression of mitochondrial ND4 leads to the protein assembling in complex I and prevents optic atrophy and visual loss
- (2015) Hélène Cwerman-Thibault et al. Molecular Therapy-Methods & Clinical Development
- LHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile
- (2014) R. Koilkonda et al. INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
- Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
- (2014) Robert E MacLaren et al. LANCET
- Trial End Points and Natural History in Patients With G11778A Leber Hereditary Optic Neuropathy
- (2014) Byron L. Lam et al. JAMA Ophthalmology
- Clinical features of MS associated with Leber hereditary optic neuropathy mtDNA mutations
- (2013) G. Pfeffer et al. NEUROLOGY
- AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
- (2012) J. Bennett et al. Science Translational Medicine
- 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
- (2010) Rajeshwari D. Koilkonda ARCHIVES OF OPHTHALMOLOGY
- Natural History of Leber's Hereditary Optic Neuropathy: Longitudinal Analysis of the Retinal Nerve Fiber Layer by Optical Coherence Tomography
- (2010) Piero Barboni et al. OPHTHALMOLOGY
- Mitochondrial optic neuropathies – Disease mechanisms and therapeutic strategies
- (2010) Patrick Yu-Wai-Man et al. PROGRESS IN RETINAL AND EYE RESEARCH
- The optimized allotopic expression of ND1 or ND4 genes restores respiratory chain complex I activity in fibroblasts harboring mutations in these genes
- (2008) Crystel Bonnet et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH
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- (2008) M Hellström et al. GENE THERAPY
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- (2008) Albert M. Maguire et al. NEW ENGLAND JOURNAL OF MEDICINE
- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
- (2008) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
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