Researchers Explore Gene Editing As a New Cure for Blood Disorders
Key TakeawaysThere is only one FDA-approved treatment for sickle cell disease, but it requires a sibling donor.Using CRISPR-CAS9 technology, researchers were successful in targeting a genetic switch that turned off production for a fetal form of hemoglobin.CRISPR-CAS9 allowed patients with sickle cell disease and beta-thalassemia to be their own donors for bone marrow transplants. This has the potential to make treatments more widely accessible. Key Takeaways There is only one FDA-approved treatment for sickle cell disease, but it requires a sibling donor....