Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates

Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset

(2021) Nancy J. Newman et al.   OPHTHALMOLOGY

Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study

(2021) Catherine Vignal-Clermont et al.   BIODRUGS

Adaptive responses to neurodegenerative stress in glaucoma

(2021) David J. Calkins   PROGRESS IN RETINAL AND EYE RESEARCH

Natural history of patients with Leber hereditary optic neuropathy—results from the REALITY study

(2021) Patrick Yu-Wai-Man et al.   EYE

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

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

Seven Years Follow-up of Gene Therapy for Leber's Hereditary Optic Neuropathy

(2020) Jiajia Yuan et al.   OPHTHALMOLOGY

Redistribution of metabolic resources through astrocyte networks mitigates neurodegenerative stress

(2020) Melissa L. Cooper et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Astrocyte remodeling without gliosis precedes optic nerve axonopathy

(2018) Melissa L. Cooper et al.   Acta Neuropathologica Communications

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

Single-cell analysis of experience-dependent transcriptomic states in the mouse visual cortex

(2017) Sinisa Hrvatin et al.   NATURE NEUROSCIENCE

AAV-Mediated Anterograde Transsynaptic Tagging: Mapping Corticocollicular Input-Defined Neural Pathways for Defense Behaviors

(2017) Brian Zingg et al.   NEURON

Gene Therapy for Leber Hereditary Optic Neuropathy

(2017) John Guy et al.   OPHTHALMOLOGY

Mechanics of mitochondrial motility in neurons

(2016) Erin L Barnhart   CURRENT OPINION IN CELL BIOLOGY

Transfer of mitochondria from astrocytes to neurons after stroke

(2016) Kazuhide Hayakawa et al.   NATURE

Gene Therapy for Leber Hereditary Optic Neuropathy

(2016) William J. Feuer et al.   OPHTHALMOLOGY

Visual BOLD Response in Late Blind Subjects with Argus II Retinal Prosthesis

(2016) E. Castaldi et al.   PLOS BIOLOGY

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

Regulation of Mitochondrial Biogenesis and Its Intersection with Inflammatory Responses

(2015) Anne D. Cherry et al.   ANTIOXIDANTS & REDOX SIGNALING

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

Fellow eye effect of unilateral intravitreal bevacizumab injection in eyes with diabetic macular edema

(2014) J Hanhart et al.   EYE

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

Astrocytes eyeball axonal mitochondria

(2014) T. C. Burdett et al.   SCIENCE

Efficient mitochondrial biogenesis drives incomplete penetrance in Leber’s hereditary optic neuropathy

(2013) Carla Giordano et al.   BRAIN

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

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