Article
An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials.
International journal of molecular sciences - 26 Apr 2021
Chiu Wei, Lin Ting-Yi, Chang Yun-Chia, Isahwan-Ahmad Mulyadi Lai Henkie, Lin Shen-Che, Ma Chun, Yarmishyn Aliaksandr A, Lin Shiuan-Chen, Chang Kao-Jung, Chou Yu-Bai, Hsu Chih-Chien, Lin Tai-Chi, Chen Shih-Jen, Chien Yueh, Yang Yi-Ping, Hwang De-Kuang
Abstract excerpt
Inherited retinal dystrophies (IRDs) are a group of rare eye diseases caused by gene mutations that result in the degradation of cone and rod photoreceptors or the retinal pigment epithelium. Retinal degradation progress is often irreversible, with clinical manifestations including color or night blindness, peripheral visual defects and subsequent vision loss. Thus, gene therapies that restore functional retinal...
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