A clinical trial has reported success in treating a genetic disease using advanced gene-editing technology. An 18-year-old patient is essentially "cured" after receiving the innovative treatment. The trial, involving the CHU Sainte-Justine, used prime editing to correct a genetic mutation.
Prime editing directly modified the patient's hematopoietic stem cells, targeting the mutation responsible for chronic granulomatous disease (CGD). Within weeks, the anomaly was corrected in a significant portion of the cells. Dr. Élie Haddad, the lead investigator, expressed optimism about the results.
The treatment avoids the risks associated with traditional bone marrow transplants. The patient's own cells are used after gene editing in the laboratory. This eliminates the possibility of rejection and potentially increases the survival rate to 100%.