Article type:
News
Estimated read time:
1 min min read
Published:
08/11/2023
UK medicine’s regulator approved the first in the world gene-editing treatment for sickle cell disease and beta thalassaemia – inherited blood disorders caused by mutations in the genes producing haemoglobin. The therapy is based on CRISPR gene editing technology and allows the production of normal blood. Until now the only long-term cure option was bone marrow transplant but this carries a risk of rejection and needs long term immunosuppression. It is approved for people with severe disease aged 12 years or older.