期刊
ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS, VOL 20, 2019
卷 20, 期 -, 页码 201-216出版社
ANNUAL REVIEWS
DOI: 10.1146/annurev-genom-083118-015043
关键词
iPSC; gene therapy; cell therapy; retinitis pigmentosa; age-related macular degeneration
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.
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