MedicineCRISPR Therapy Gets U.K. Approval, the First in the World
Britain's medicines regulator has approved the world's first gene therapy for sickle cell disease, offering hope to thousands of patients.
Casgevy is the first treatment licensed using the gene editing tool CRISPR, and it targets the problematic gene in a patient's bone marrow stem cells.
Both sickle cell disease and thalassemia are caused by genetic mutations that affect the production of hemoglobin in red blood cells. [ more ]