An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials

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 bl...

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Veröffentlicht in:Int J Mol Sci
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI 2021
Schlagworte:
Review
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8123696/
https://ncbi.nlm.nih.gov/pubmed/33926102
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22094534
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