Ten children with an especially rare and hard-to-treat form of “bubble boy” disease are living normal lives after receiving a new gene therapy approach, researchers say.
Experts said the findings are a major advance for children with the disease — a subtype of severe combined immunodeficiency (SCID).
SCID refers to a group of rare genetic diseases that cause babies to be born without a functioning immune system. It became widely known as “bubble boy” disease in the 1970s and ’80s, due to the case of David Vetter, a boy with SCID who lived in a plastic, germ-free bubble for 12 years.
