Early clinical trial data show gene therapy reversing sickle cell anemia

(Cincinnati Children's Hospital Medical Center) After over a decade of preclinical research and development, a new gene therapy treatment for Sickle Cell Anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented Dec. 3 at the American Society of Hematology's (ASH) annual meeting in San Diego.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news