CRISPR Edits Down Syndrome: Hope and Ethical Concerns Arise
Researchers at Mie University have demonstrated a CRISPR-Cas9 method to remove the extra chromosome 21 in cells from individuals with Down syndrome. This allele-specific editing successfully normalized gene expression, improving cell survival and function in both stem cells and mature skin fibroblasts. The corrected cells exhibited reduced oxidative stress and increased growth rates, suggesting alleviation of the biological burden of trisomy 21. While still in early stages, this research offers a promising avenue for future therapies targeting the root cause of Down syndrome.
John Q. Hosedrinker