Gene therapy for Leber hereditary optic neuropathy
10.3760/cma.j.cn511434-20220127-00049
- VernacularTitle:Leber遗传性视神经病变的基因治疗
- Author:
Qingmei MIAO
1
;
Changzheng CHEN
;
Jiajia YUAN
Author Information
1. 武汉大学人民医院眼科中心, 武汉 430060
- Keywords:
Leber hereditary optic neuropathy;
DNA, mitochondrial;
Adeno-associated virus;
Gene therapy;
Review
- From:
Chinese Journal of Ocular Fundus Diseases
2022;38(12):1031-1034
- CountryChina
- Language:Chinese
-
Abstract:
Leber 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.
