AI Article Synopsis
- Retinal degenerative diseases lead to significant vision loss and are a common cause of blindness.
- Gene therapy for the retina has seen major advancements, including the first FDA-approved treatment for genetic eye conditions.
- CRISPR/Cas9 technology is revolutionizing gene therapy approaches, and this review covers the evolution and future potential of these treatments for retinal diseases.
Article 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.
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Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8982485 | PMC |
| http://dx.doi.org/10.1093/pcmedi/pby004 | DOI Listing |
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