Gene therapy for retinal degeneration.
Gene therapy for retinal degeneration.
复制标题
视网膜变性的基因治疗。
DOI：
10.1159/000268024
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发表时间：
1997
影响因子：
2.1
通讯作者：
S. Bhattacharya
中科院分区：
文献类型：
--
作者：
M. Reichel;R. Ali;D. Hunt;S. Bhattacharya
遗传性视网膜变性是一组通过许多患者的视力逐渐丧失而导致失明的疾病。尽管随着越来越多的疾病基因的克隆，这些病症的分子遗传学和作为常见疾病机制的细胞凋亡途径的知识正在稳步增加，但仍然没有治愈那些受影响的人。近年来，通过许多研究人员的努力，新的实验性治疗方法已经发展出来，并在动物模型中进行了探索。本文将概述和讨论不同的策略，用于开发一种基于基因替代或拯救的患病神经元组织与生长因子的治疗。
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.