The research status and progress of gene therapy for X-linked juvenile retinoschisis
10.3760/cma.j.cn511434-20210816-00439
- VernacularTitle:X连锁视网膜劈裂症的基因治疗研究现状与进展
- Author:
Tianlu ZHANG
1
;
Yin SHEN
Author Information
1. 武汉大学人民医院眼科中心 430060
- Keywords:
Retinoschisis;
Gene therapy;
Review
- From:
Chinese Journal of Ocular Fundus Diseases
2021;37(11):891-895
- CountryChina
- Language:Chinese
-
Abstract:
X-linked juvenile retinoschisis (XLRS) is a rare X-linked inherited retinal disorder, mainly affects bilateral retina. Patients often present with visual deterioration accompanied by a spoke-wheel pattern in the macula due to splitting of inner retinal layers and a disproportionate decline in the b-wave relative to a-wave of electroretinogram. The current therapy is mainly directed toward treatment of complications with no effective clinical management yet. In recent years, with the deepening understanding of XLRS, adeno-associated virus(AAV)-mediated gene therapy has become a potential new approach for the treatment. Two clinical trials on XLRS gene therapy are currently underway. These two clinical trials assess the ocular safety and tolerability of recombinant AAV- RS1 vector and explore its safe dose in XLRS patients. However, the recovery of retinal structure and function in XLRS patients is unsatisfactory. Following the in-depth research and progress of clinical trials, it is expected that more accurate and effective treatments for XLRS patients will be provided in the future.
