Efficacy and Safety of rAAV2-ND4 Treatment for Leber’s Hereditary Optic Neuropathy

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

Safety and Effects of the Vector for the Leber Hereditary Optic Neuropathy Gene Therapy Clinical Trial

(2014) Rajeshwari D. Koilkonda et al.   JAMA Ophthalmology

Leber's hereditary optic neuropathy masquerading as optic neuritis with spontaneous visual recovery

(2013) Tsui-Kang Hsu et al.   Clinical and Experimental Optometry

Effect of EPI-743 on the Clinical Course of the Mitochondrial Disease Leber Hereditary Optic Neuropathy

(2012) Alfredo A. Sadun   ARCHIVES OF NEUROLOGY

Adeno-associated virus-mediated gene delivery of the human ND4 complex I subunit in rabbit eyes

(2012) Hui Shi et al.   CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Applications of the method of high resolution melting analysis for diagnosis of Leber's disease and the three primary mutation spectrum of LHON in the Han Chinese population

(2012) Guanglin Cui et al.   GENE

Effects of Idebenone on Color Vision in Patients With Leber Hereditary Optic Neuropathy

(2012) Guenther Rudolph et al.   JOURNAL OF NEURO-OPHTHALMOLOGY

Treatment of Leber hereditary optic neuropathy

(2011) N. J. Newman   BRAIN

Idebenone Treatment In Leber's Hereditary Optic Neuropathy

(2011) V. Carelli et al.   BRAIN

A randomized placebo-controlled trial of idebenone in Leber’s hereditary optic neuropathy

(2011) Thomas Klopstock et al.   BRAIN

Oestrogens ameliorate mitochondrial dysfunction in Leber’s hereditary optic neuropathy

(2010) Carla Giordano et al.   BRAIN

Adeno-associated Virus Vectors Serotype 2 Induce Prolonged Proliferation of Capsid-Specific CD8+ T Cells in Mice

(2010) Hua Li et al.   MOLECULAR THERAPY

Mitochondrial optic neuropathies – Disease mechanisms and therapeutic strategies

(2010) Patrick Yu-Wai-Man et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Optimized Allotopic Expression of the Human Mitochondrial ND4 Prevents Blindness in a Rat Model of Mitochondrial Dysfunction

(2008) Sami Ellouze et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(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