The gene therapy that has seemingly cured a 2-year-old girl could be used to treat other diseases, like Sickle Cell Anemia.

UCLA DR. DONALD KHON has perfected the treatment that has now affected the lives of over a dozen families, including the parents of Evangelina, who was born with the deadly SCID. The immune system disease is known by its common name, Bubble Baby disease, because the children have to live in bubbles, since their immune systems do not function. Most die within the first year of life, unless they can get and survive a bone marrow transplant.

Evangelina's twin sister, Annabella, was not a bone marrow transplant. So Dr Kohn included the sick twin in his study, transfering the patient's own bone marrow to the child. At two, her immune system is working well.

It's a difficult and time consuming process, but it seems to work. The same procedure could work with other blood diseases that are curable with bone marrow transfusions. That includes sickle cell anemia, which will be the focus of the next trial.

The implications are huge, as there are 30 or 40 diseases where the treatment could work.

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Bubble Baby: Gene Therapy Cured 2-year-old Girl Of Deadly SCID At UCLA