Study: Gene treatment benefits children with rare immune disease

01/28/2009 | NYTimes.com

A new study found that eight of 10 children with an uncommon form of severe combined immunodeficiency -- a rare genetic disorder that impairs the body's ability to fight infections, also called "bubble boy disease" -- stopped taking medications about four years after being injected with a gene treatment. None of the patients developed leukemia or other complications from the treatment, researchers reported. The therapy combines bone marrow cells from patients with functional copies of the gene for an enzyme called adenosine deaminase that is lacking in people with the condition.

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