Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling illness in the U.K.
I’ve been waiting for this ever since I learned about CRISPR! Knowing that sickle cell anemia is the result of a single point mutation, I always figured it would be one of the first diseases cured by gene therapy.
I’ve been waiting for this ever since I learned about CRISPR! Knowing that sickle cell anemia is the result of a single point mutation, I always figured it would be one of the first diseases cured by gene therapy.