The progress in treatment of type 1 of Stargardt disease

VernacularTitle:Stargardt病1型的治疗研究现状与进展
Author: Dandan WANG ¹ ; Fengjuan GAO ; Shenghai ZHANG ; Qing CHANG ; Gezhi XU ; Jihong WU
Author Information

1. 复旦大学附属眼耳鼻喉科医院眼科研究院，上海　200031
Keywords: Abca4 gene; Stargardt disease; Drug therapy; Gene therapy; Stem cell transplantation; Review
From: Chinese Journal of Ocular Fundus Diseases 2021;37(7):567-572
CountryChina
Language:Chinese
Abstract: Stargardt disease (STGD) is one of the most prevalent inherited macular dystrophy, and most often occurs in child or adolescence. Irreversible vision loss is observed in almost all cases. Type 1 (STGD1) is one of the most common type. It is an autosomal recessive condition, caused by mutations in the Abca4 gene. In recent years, encouraging progress has been made in the treatment of STGD1. C20-D3-retinyl acetate (ALK- 001), fenretinide and ICR-14967 (A1120) as visual cycle modulators, StarGen as gene supplementation therapies, and the stem cell transplantation of human embryonic stem cell-derived retinal pigment epithelium cells are the most promising therapies. With the development of studies and clinical trials, the clinical application of various treatments of STGD1 are expected in the near feature, which are expected to save the vision of most patients.