Open AccessMultilocus Mitochondrial Mutations Do Not Directly Affect the Efficacy of Gene Therapy for Leber Hereditary Optic Neuropathy
Author(s) -
Shuo Yang,
Chen Chen,
Jiajia Yuan,
Shuaishuai Wang,
Xing Wan,
Heng He,
Siqi Ma,
Bin Li
Publication year - 2020
Publication title -
journal of neuro-ophthalmology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.586
H-Index - 55
eISSN - 1536-5166
pISSN - 1070-8022
DOI - 10.1097/wno.0000000000000797
Subject(s) - mitochondrial dna , genetic enhancement , nonsynonymous substitution , mutation , genetics , optic neuropathy , gene , leber's hereditary optic neuropathy , mitochondrial disease , visual acuity , biology , clinical trial , gene mutation , mitochondrion , medicine , bioinformatics , ophthalmology , genome , optic nerve
Clinical trials of gene therapy for Leber hereditary optic neuropathy (LHON) were conducted in 9 volunteers with the mitochondrial mutation, G11778A in ND4. The purpose of this study was to investigate whether multilocus mitochondrial mutations directly influence the efficacy of gene therapy for LHON.