Researchers have successfully restored vision in adults and children with a rare form of inherited blindness using CRISPR-Cas9 gene editing technology. This breakthrough, published in The New England Journal of Medicine, offers promise for treating other genetic disorders.
The BRILLIANCE Trial:
- Participants: 14 individuals (12 adults, 2 children) with Leber congenital amaurosis (LCA) - a genetic condition causing severe vision loss.
- Treatment: Single dose of EDIT-101, a CRISPR gene therapy targeting the CEP290 gene mutation responsible for LCA.
- Results:
- 11 out of 14 participants experienced improved vision, including the ability to see colors, navigate, and read basic charts.
- No major side effects reported.
CRISPR-Cas9 Technology:
- Allows for precise editing of DNA by removing and inserting specific strands.
- Holds great promise for treating a wide range of genetic diseases beyond blindness.
Future of Gene Editing:
CRISPR technology is rapidly advancing, with ongoing clinical trials exploring its potential for treating HIV/AIDS, diabetes, cancer, and other conditions. This breakthrough paves the way for a new era of gene-based therapies.
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