Featured Article Academic Journal Main Category: HIV / AIDS Also Included In: Clinical Trials / Drug Trials Article Date: 16 Jul 2013 - 0:00 PDT Ad For Health Professionals

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The HIV Virus can be used to treat two severe genetic diseases, according to two studies published in the journal Science.

Researchers from the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan, Italy, have revealed that the HIV virus can be used to treat metachromatic leukodystrophy, a disease impairing the fatty covering that acts as an insulator around nerve fibers and Wiskott-Aldrich Syndrome, an immune system deficiency reducing the ability to form blood clots.

The researchers say that the origin of both these diseases is a genetic defect that results in the deficiency of a protein essential in the early years of life. The HIV therapy technique, called lentiviral hematopietic stem cell gene therapy, involves withdrawing hematopoietic stem cells - cells isolated from the bone marrow that can renew themselves - from the bone marrow of the patients.

The patients are then given a corrected copy of the defected gene using viral vectors derived from HIV. A viral vector is a tool that enables the transfer of genetic material into cells. Once this process is complete, the treated cells are able to restore the missing proteins to the key organs, researchers say.

The results of the study revealed that six children from all over the world have received this treatment over the last three years, and are "well and show significant benefits."

Alessandro Aiuti, head of research of the pediatric clinic at TIGET, explains:

"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. Thanks to gene therapy, the children no longer have to face severe bleeding and infection. They can run, play and go to school.

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HIV Virus Used As Vector To Get Gene Therapy Into Six Children

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