Gene editing therapy leads to a huge win for sickle cell patients
New England Journal of Medicine | Rabi Hanna, Haydar Fangoul, et al. About 500,000 children worldwide are born with sickle cell disease each year. The disease causes health complications, including extreme pain. But 27 of 28 patients who received an experimental gene therapy that modified their own blood-forming stem cells had no painful episodes after the treatment. The therapy corrects the mutation that causes red blood cells to become sickle-shaped. *Note: Some users may encounter a paywall beyond the abstract. (Read)
