A clinical trial for a new gene therapy approach to treat sickle cell disease has been approved to proceed by the US Food and Drug Administration. Patients with sickle cell disease have a mutation in the β -haemoglobin gene, causing them to produce misshapen red blood cells that can block blood vessels leading to severe pain, anaemia and potentially life-threatening complications. In the newly-approved trial, scientists from the University of California will use CRISPR/Cas9 genome editing to replace the faulty gene with a functional version.
