Seven-Year Follow-up of Gene Therapy for Leber’s Hereditary Optic Neuropathy

作者：Jiajia Yuan, Yong Zhang, Hongli Liu, Dan Wang, Yangyang Du, Zhen Tian, Xin Li, Shuo Yang, Han Zhong Pei, Xing Wan, Su Xiao, Lin Song, Xiao Xiao, Jian Sun, Zhitao Wang, Bin Li · 发表于：Ophthalmology · 年份：2020 · DOI：10.1016/j.ophtha.2020.02.023 · 被引用次数：59 · 研究领域：Retinal Development and Disorders、Mitochondrial Function and Pathology、Photosynthetic Processes and Mechanisms

Leber’s hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial mutation with simultaneous or consecutive binocular painless loss of vision and central visual field (VF) defects.1 In 2008, we initiated recombinant adeno-associated virus-mediated gene therapy research for LHON. Between 2011 and 2012, we evaluated the efficacy, durability, and long-term safety in 9 patients with the LHON mt11778G→A mutation (ClinicalTrials.gov identifier, NCT01267422). Except for patient 1, all patients received a single intravitreal injection of gene therapy agent.