Zhorzholadze N V, Sheremet N L, Tanas A S, Strelnikov V V
Research Institute of Eye Diseases, Moscow, Russia.
Research Centre for Medical Genetics, Moscow, Russia.
Vestn Oftalmol. 2020;136(4. Vyp. 2):333-343. doi: 10.17116/oftalma2020136042333.
Stargardt disease is a hereditary retinal dystrophy associated with mutations in the gene. Currently, no etiopathogenetic drugs nor treatment methods for Stargardt disease have completely passed clinical trials. The review summarizes experimental and clinical studies of drugs aimed at reducing the accumulation of vitamin A dimers, lipofuscin, complement inhibition and RPE regeneration by stem cell transplantation, as well as gene therapy studies with intravitreal vector injection of the functional gene.
斯塔加特病是一种与该基因突变相关的遗传性视网膜营养不良。目前,尚无针对斯塔加特病的病因发病机制药物或治疗方法完全通过临床试验。本综述总结了旨在通过干细胞移植减少维生素A二聚体、脂褐质积累、补体抑制和视网膜色素上皮(RPE)再生的药物的实验和临床研究,以及通过玻璃体内注射功能性基因载体进行基因治疗的研究。