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…
Scientists Discover How Gene Mutation Triggers Immune Disease
Scientists discovered how a gene mutation affects T cell function to promote immune disorders and then tested a treatment based on the discovery–successfully fixing donated immune cells from a 16-year-old boy with an abnormally low level of white blood cells called lymphopenia. Cincinnati Children’s researchers report their findings Jan. 30 in Nature Communications. The discovery…
