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Gene and Induced Pluripotent Stem Cell Therapy for Retinal Diseases.
Annual Review of Genomics and Human Genetics ( IF 7.7 ) Pub Date : 2019-04-24 , DOI: 10.1146/annurev-genom-083118-015043
Akiko Maeda 1 , Michiko Mandai 1 , Masayo Takahashi 1, 2
Affiliation  

Given the importance of visual information to many daily activities, retinal degenerative diseases-which include both inherited conditions (such as retinitis pigmentosa) and acquired conditions (such as age-related macular degeneration)-can have a dramatic impact on human lives. The therapeutic options for these diseases remain limited. Since the discovery of the first causal gene for retinitis pigmentosa almost three decades ago, more than 250 genes have been identified, and gene therapies have been rapidly developed. Simultaneously, stem cell technologies such as induced pluripotent stem cell-based transplantation have advanced and have been applied to the treatment of retinal degenerative diseases. Here, we review recent progress in these expanding fields and discuss the potential for precision medicine in ophthalmic care.

中文翻译:

基因和诱导多能干细胞疗法治疗视网膜疾病。

考虑到视觉信息对于许多日常活动的重要性,视网膜变性疾病-既包括遗传性疾病(例如色素性视网膜炎)又包括后天性疾病(例如与年龄有关的黄斑变性),可能对人类生活产生重大影响。这些疾病的治疗选择仍然有限。自从大约三十年前发现第一个色素性视网膜炎的致病基因以来,已鉴定出250多个基因,并且基因疗法迅速发展。同时,诸如基于诱导多能干细胞的移植等干细胞技术已经发展,并已被用于治疗视网膜退行性疾病。在这里,我们回顾了这些扩展领域的最新进展,并讨论了精密医疗在眼科护理中的潜力。
更新日期:2020-04-21
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