Science & Technology


X-reactivation gene therapy is a novel approach to correcting X-linked dominant genetic mutations in females. When a mutation is X-linked, some of the patient’s cells express a healthy, functional copy of the gene (from an unaffected parent), while other cells express the damaged gene (from the affected parent). Traditional gene replacement therapy would deliver functional gene to all cells, resulting in overexpression of the gene in the healthy cells. To avoid this, X-reactivation instead re-activates the silenced, healthy X chromosome that exists within the affected cells, restoring expression in the impacted cells while leaving the normal cells unchanged.