-- Robert Preidt
THURSDAY, July 11 (HealthDay News) -- Turning a medical foe into
a therapeutic friend, Italian scientists say they used a piece of
HIV to cure two rare genetic diseases affecting children.
HIV is the virus that causes AIDS.
Gene therapy using the technique proved effective in three
children with metachromatic leukodystrophy and three others with
Wiskott-Aldrich syndrome. After three years of treatment, all of
them are now well and show significant benefits from the
"The results obtained from the first six patients are very encouraging: The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases," study lead researcher Luigi Naldini, director of the San Raffaele Telethon Institute for Gene Therapy (TIGET), in Milan, said in an institute news release.
The cause of both diseases is a genetic defect that results in
the deficiency of a protein essential in the early years of life.
In the case of metachromatic leukodystrophy, the nervous system is
affected. Babies with this previously incurable disease appear
healthy at birth, but at some point they begin to gradually lose
the thinking and physical skills they have developed, with no
possibility of halting the decline.
Children with Wiskott-Aldrich syndrome have a faulty immune
system that makes them much more vulnerable than normal to the
development of infections, autoimmune diseases and cancer. They
also have a defect in the platelets, which causes frequent
In this new therapy, stem cells are taken from the bone marrow
of the patient. But a viral vector -- a means of transporting an
agent into a cell -- is needed to transport a corrected copy of the
gene that is defective into the stem cells. The Italian team
obtained just such a viral vector from HIV.
Once reinjected into the body, the treated stem cells are able
to restore the missing protein to key organs, the researchers
"In patients with Wiskott-Aldrich syndrome, blood cells are directly affected by the disease and the corrected stem cells replace the diseased cells, creating a properly functioning immune system and normal platelets," said Alessandro Aiuti, head of research of the Pediatric Clinic at TIGET. "Thanks to gene therapy, the children no longer have to face severe bleeding and infection. They can run, play and go to school."
"In the case of metachromatic leukodystrophy, however, the therapeutic mechanism is more sophisticated: The corrected [stem] cells reach the brain through the blood and release the correct protein that is 'gathered' there by the surrounding nerve cells," colleague Alessandra Biffi said in the news release.
The studies were published July 11 in the journal
The U.S. National Institute of Neurological Disorders and Stroke
has more about
metachromatic leukodystrophyand the U.S. National
Library of Medicine has more about
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