Gene Therapy Keeps “Bubble Boy” Disease at Bay in 8 Children
December 07, 2013
Researchers say they are achieving success in curing the genetic
defect that causes some children to be born without immune defenses, a rare
condition made famous in the 1970s by a Texas boy who lived most of his short
life in a sterile "bubble,” NPR reports. Scientists now report that 8 out
of 9 young children given gene therapy for a type of severe combined
immunodeficiency disease, called SCID-X1, are alive and living amid the
everyday microbial threats that would otherwise have killed them.
the full story. Read the news release.