Gene therapy cures two men of immune system disorder

doi:10.1136/bmj.332.7545.812

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Annette Tuffs

Heidelberg

A multinational team of researchers led by Germans has reported the first successful use of gene therapy to treat two adult patients with an immunesystem disorder that could not otherwise be treated.

The researchers' study was published online ahead of print publication on 2 April in Nature Medicine (www.nature.com/nm, doi:10.1038/nm1393). The researchers—from Germany, the United Kingdom, United States, and Switzerland—said that they were able to cure the two men, aged 25 and 16, who had the genetic disorder chronic granulomatous disease (CGD). The illness results form …