Tag Archives: gene therapies

Promising new gene therapies for sickle cell remain unavailable in countries where they are needed most

Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. Now that new gene therapies promise a cure for sickle cell disease, Dongre says he’s “praying for the cure to come to us.” But experts say a one-time treatment… Read More »

FDA approves 2 gene therapies for sickle cell. One of the first to use the CRISPR editing tool

Regulators approved two gene therapies for sickle cell disease on Friday. Doctors hope they may be able to cure painful, inherited blood disease that mostly affects black people in the US The Food and Drug Administration said the one-time treatments can be used for patients ages 12 and older with severe forms of the disease.… Read More »