Gene therapy superior to half-matched transplant for children with 'bubble boy disease'

New research published online in Blood , the Journal of the American Society of Hematology , reports that children with "bubble boy disease" who undergo gene therapy have fewer infections and hospitalizations than those receiving stem cells from a partially matched donor. The research is the first to compare outcomes among children with the rare immune disorder - also known as X-linked severe combined immunodeficiency - receiving the two therapeutic approaches.

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