Gene therapy cures two men of immune system disorder

doi:10.1136/bmj.332.7545.812

BMJ 2006;332:812 (8 April), doi:10.1136/bmj.332.7545.812

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Gene therapy cures two men of immune system disorder

Heidelberg Annette Tuffs

A multinational team of researchers led by Germans has reported the first successful use of gene therapy to treat two adult patients with an immune system 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 an inherited defective gene on the maternal X chromosome. It makes patients highly susceptible to infections, because defective phagocytes are incapable of destroying harmful bacteria and fungi.

The two men suffered constant, life threatening infections because of their illness. The only treatment for people with CGD was a bone marrow transplantation to replace the defective blood cells. . . . [Full text of this article]

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