CRISPR-Cas9

Louisiana Man First Functionally Cured of Sickle Cell Disease

A 23-year-old New Orleans man has become the first in Louisiana to be functionally cured of sickle cell disease through gene therapy. This innovative treatment, which involves editing a patient’s own stem cells, was performed at Manning Family Children’s Hospital. Following this life-changing two-year journey, he aims to establish a nonprofit to support others facing similar challenges, emphasizing equitable access to treatment for all Louisianans affected by the disease.

Read More

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.

Read More