Reichel M B, Ali R R, Hunt D M, Bhattacharya S S
Department of Ophthalmology, Faculty of Medicine, University of Leipzig, Deutschland.
Ophthalmic Res. 1997;29(5):261-8. doi: 10.1159/000268024.
Inherited retinal degenerations are a group of diseases leading to blindness through progressive loss of vision in many patients. Although with the cloning of more and more disease genes the knowledge on the molecular genetics of these conditions and on the apoptotic pathway as the common disease mechanism is steadily increasing, there is still no cure for those affected. In recent years, new experimental treatments have evolved through the efforts of many investigators and have been explored in animal models. The rationale of the different strategies for developing a treatment based on gene replacement or rescue of the diseased neuronal tissue with growth factors will be outlined and discussed in this paper.
遗传性视网膜变性是一组疾病，许多患者会因视力逐渐丧失而失明。尽管随着越来越多疾病基因的克隆，人们对这些病症的分子遗传学以及作为常见疾病机制的凋亡途径的了解在不断增加，但对于患者仍然没有治愈方法。近年来，通过许多研究人员的努力，新的实验性治疗方法不断涌现，并已在动物模型中进行了探索。本文将概述并讨论基于基因替代或用生长因子挽救患病神经组织来开发治疗方法的不同策略的基本原理。
