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 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.

SUBMITTER: Fu X 

PROVIDER: S-EPMC8982485 | biostudies-literature | 2018 Jun

REPOSITORIES: biostudies-literature