摘要Leber遗传性视神经病变（LHON）是一种母系遗传性视神经疾病，由线粒体DNA突变导致视网膜神经节细胞退行性改变。目前该病治疗手段有限，早期进行对症支持治疗以及药物治疗可能对患者视力有所改善。基因治疗近年来进展迅速，多项临床研究已证实其安全性和有效性。单眼基因治疗对LHON患者视功能改善有一定帮助，且可以提高未注射眼视力，安全性和耐受性较好。未来需要更大样本量、更长随访时间的研究来进一步验证基因治疗的疗效及安全性。基因治疗有望成为一种安全有效的治疗方式，为LHON患者带来希望。

abstractsLeber hereditary optic neuropathy (LHON) is a matrilineal hereditary optic neuropathy in which mitochondrial DNA mutations lead to retinal ganglion cell degeneration. At present, the treatment for LHON is limited. Early symptomatic treatment and medical treatment may improve the vision of patients. In recent years, rapid progress has been made in gene therapy. Many clinical studies have confirmed its safety and efficacy. Monocular gene therapy is helpful to improve the visual function of LHON patients, and it can also improve the visual acuity of uninjected eyes. Patients do not have serious eye or systemic adverse events during the treatment period, showing good safety and tolerance. Studies with larger sample size and longer follow-up time are needed to further verify the efficacy and safety of gene therapy in the future. Gene therapy is expected to become a safe and effective treatment, bringing hope to LHON patients.