LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: Biodistribution and toxicology profile

Investigative Ophthalmology and Visual Science

Volumn 55, Issue 12, 2014, Pages 7739-7753

  Koilkonda, Rajeshwari ^a   Yu, Hong ^a   Talla, Venu ^a   Porciatti, Vittorio ^a   Feuer, William J ^a   Hauswirth, William W ^b   Chiodo, Vince ^b   Erger, Kirsten E ^b   Boye, Sanford L ^b   Lewin, Alfred S ^b   Conlon, Thomas J ^b   Renner, Lauren ^c   Neuringer, Martha ^c   Detrisac, Carol ^d   Guy, John ^a  

^a UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^c OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^d Charles River Pathology Associates   (United States)

Author keywords

Gene therapy; LHON; Mitochondria

Indexed keywords

MITOCHONDRIAL DNA; NADH DEHYDROGENASE SUBUNIT 4; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE DEHYDROGENASE;

ADVERSE EFFECTS; ANIMAL; BLINDNESS; DISEASE MODEL; ELECTRORETINOGRAPHY; GENE THERAPY; GENE VECTOR; GENETICS; METABOLISM; MITOCHONDRION; MOUSE; NERVE FIBER; OPTIC ATROPHY, HEREDITARY, LEBER; OPTICAL COHERENCE TOMOGRAPHY; PATHOPHYSIOLOGY; PROCEDURES; RAT; RETINA GANGLION CELL; RHESUS MONKEY; TRANSMISSION ELECTRON MICROSCOPY; ULTRASTRUCTURE;

ANIMALS; AXONS; BLINDNESS; DISEASE MODELS, ANIMAL; DNA, MITOCHONDRIAL; ELECTRORETINOGRAPHY; GENETIC THERAPY; GENETIC VECTORS; MACACA MULATTA; MICE; MICROSCOPY, ELECTRON, TRANSMISSION; MITOCHONDRIA; NADH DEHYDROGENASE; OPTIC ATROPHY, HEREDITARY, LEBER; RATS; RETINAL GANGLION CELLS; TOMOGRAPHY, OPTICAL COHERENCE;

EID: 84914166610     PISSN: 01460404     EISSN: 15525783     Source Type: Journal    
DOI: 10.1167/iovs.14-15388     Document Type: Article

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