Scientists find unlikely comrade in gene therapy for fatal disease
By The Associated Press
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Published: November 6, 2009
WASHINGTON — French scientists mixed gene therapy and bone marrow transplants in two boys to seemingly halt a brain disease that can be fatal by adolescence.
The surprise ingredient: They disabled the HIV virus so it couldn’t cause AIDS, and then used it to transport the healthy new genetic material.
The experiment marks the first time researchers have tried that long-contemplated step in people — and the first effective gene therapy against a severe brain disease, said lead researcher
Dr. Patrick Aubourg of the University Paris-Descartes.
Although it’s a small, first-step study, it has "exciting implications” for other blood and immune disorders that had been feared beyond gene therapy’s reach, said
Dr. Kenneth Cornetta, president of the
American Society of Gene and Cell Therapy.
Adrenoleukodystrophy, or ALD, is a rare genetic disease that, in its most devastating form, destroys the coating of nerve fibers in boys’ brains. Without that coating, called myelin, the neurological system breaks down. The disease often strikes between the ages of four and 10.
Two years later, the boys have shown no sign of worsening brain damage and are functioning well with 15 percent of their blood cells producing the healthy protein, said Aubourg.
Related Topics:
Science and Technology,
Technology,
Health and Fitness,
Sexual and Reproductive Health,
HIV and AIDS,
Sexually Transmitted Diseases,
Contagious and Infectious Diseases,
Life Sciences,
Biology,
Medical Technology,
Genetics,
Biotechnology,
Gene Therapy
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