Pioneering New Therapies for Pediatric Patients

A phase 1 clinical trial of a treatment for severe sickle cell disease (SCD) using gene therapy opened at MUSC in 2015. In SCD a mutation in the beta-globin gene causes the normally round and flexible red blood cells to take on a rigid sickle shape that make it difficult for them to pass through small blood vessels.

More at Progressnotes, MUSC’s Medical Magazine