Six patients with rare blood disease are doing well after gene therapy clinical trial

(University of California - Los Angeles Health Sciences) UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD - which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan -- had to rely on bone marrow donations for a chance at remission.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news