Researchers at CHOP and the University of Pennsylvania Perelman School of Medicine pioneered a new model that offers a potential platform for developing novel therapies to treat Alpha Thalassemia.
Researchers developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.
Stefano Rivella, PhD, is developing a novel gene therapy that could help patients living with sickle cell disease and other debilitating red blood disorders.