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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Detalhes bibliográficos
Publicado no:Int J Mol Sci
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2021
Assuntos:
Review
Acesso em linha: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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