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Purpose:The aim of this study was to establish a fast,simple,and accurate mutation-detection approach to select patients with Leber Hereditary Optic Neuropathy (LHON) and carrying a mutation at nucleotide position 11778 in the mitochondrial DNA (mtDNA) but no other mitochondrial mutations for AAV vector-delivered NADH-ubiquinone oxidoreductase,subunit 4 (AAV2-ND4) gene therapy.