Clinical applications of retinal gene therapies

Abstract 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 Repe...

Full description

Saved in:
Bibliographic Details
Published in:Precision clinical medicine 2018-06, Vol.1 (1), p.5-20
Main Authors: 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
Format: Article
Language:English
Subjects:
Review
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Abstract 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.
ISSN:2516-1571
2096-5303
2516-1571
DOI:10.1093/pcmedi/pby004