Open AccessLHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile
Author(s) -
Rajeshwari D. Koilkonda,
Hong Yu,
Venu Talla,
Vittorio Porciatti,
William J. Feuer,
William W. Hauswirth,
Vince A. Chiodo,
Kirsten Erger,
Sanford L. Boye,
Alfred S. Lewin,
Thomas J. Conlon,
Lauren Renner,
Martha Neuringer,
Carol J. Detrisac,
John Guy
Publication year - 2014
Publication title -
investigative ophthalmology and visual science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.935
H-Index - 218
eISSN - 1552-5783
pISSN - 0146-0404
DOI - 10.1167/iovs.14-15388
Subject(s) - optic neuropathy , mitochondrial dna , microbiology and biotechnology , biology , optic nerve , pathology , medicine , anatomy , genetics , gene
To demonstrate safety and efficacy of allotopic human ND4 for treatment of a Leber's hereditary optic neuropathy (LHON) mouse model harboring the G11778A mitochondrial mutation.