Scientists Halt Brain Disease With New Gene Therapy

More recently, though, doctors have made encouraging steps. A study published in October showed success with gene therapy in a type of inherited blindness call Leber congenital amaurosis [ID:nN24142112] and in January a follow-up study of SCID children concluded that eight of 10 treated seemed to be cured.

In their study, Aubourg and his team took blood stem cells from the patients' bone marrow and used the new vector system to genetically alter them by inserting a working copy of the ALD gene. The modified cells were then put back into the patients.

Two years later, the scientists still detected ALD proteins in the patients, and the disease had stopped getting worse.

Aubourg said he expected this gene approach could be used in future for many more patients needing bone marrow transplants.

(Editing by Elizabeth Fullerton)

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