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Article

Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.

JAMA ophthalmology - 1 Apr 2014

Koilkonda Rajeshwari D, Yu Hong, Chou Tsung-Han, Feuer William J, Ruggeri Marco, Porciatti Vittorio, Tse David, Hauswirth William W, Chiodo Vince, Boye Sanford L, Lewin Alfred S, Neuringer Martha, Renner Lauren, Guy John

Abstract excerpt

IMPORTANCE We developed a novel strategy for treatment of Leber hereditary optic neuropathy (LHON) caused by a mutation in the nicotinamide adenine dinucleotide dehydrogenase subunit IV (ND4) mitochondrial gene. OBJECTIVE To demonstrate the safety and effects of the gene therapy vector to be used in a proposed gene therapy clinical trial. DESIGN AND SETTING In a series of laboratory experiments, we modified the...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 24457989
DOI: 10.1001/jamaophthalmol.2013.7630
PMCID: PMC4266107

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