In a major step toward treating rare neurological diseases, scientists have used gene editing to correct mutations inside the brain of living mice with a single injection. This new approach, described ...
A team of researchers at Columbia, Harvard and University of Minnesota has developed a gene editing technique they say could pave the way for new therapies for a host of diseases with fewer unintended ...
A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...