Clinical applications of retinal gene therapies

Retinal degenerative diseases are a major cause of blindness. Retinal gene therapy is a trail-blazer in the human gene therapy field, leading to the first FDA approved gene therapy product for a human genetic disease. The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9 (...

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Detalles Bibliográficos
Autores principales: Fu, Xin, Huu, Viet Anh Nguyen, Duan, Yaou, Kermany, Daniel S, Valentim, Carolina C S, Zhang, Runze, Zhu, Jie, Zhang, Charlotte L, Sun, Xiaodong, Zhang, Kang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2018
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8982485/
https://www.ncbi.nlm.nih.gov/pubmed/35694125
http://dx.doi.org/10.1093/pcmedi/pby004
Descripción
Sumario:Retinal degenerative diseases are a major cause of blindness. Retinal gene therapy is a trail-blazer in the human gene therapy field, leading to the first FDA approved gene therapy product for a human genetic disease. The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9 (CRISPR/Cas9)-mediated gene editing technology is transforming the delivery of gene therapy. We review the history, present, and future prospects of retinal gene therapy.

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