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

Researchers have 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.

from Health & Medicine News -- ScienceDaily https://www.sciencedaily.com/releases/2020/01/200129091403.htm