Harvard gene editing treatment corrects brain mutations and improves survival

Harvard gene editing treatment corrects brain mutations and improves survival

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 in the journal Cell, could reshape the future for people with devastating conditions once thought untreatable. The disorder targeted in the study is called alternating hemiplegia of childhood (AHC), a disease that strikes in infancy and causes sudd…