9 babies healed by Italian gene therapy

(Stem Cells News image)

Gene therapy using an Adenovirus vector. A new...
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It has been about 8 years since Salsabil, the first Palestinia baby suffering congenital immunodeficiency (Ada-Scid), has been healed using gene therapy. And it has been about 7 years since her story to Abdul Rahim’s one, a Pakistani baby born in the end of 2006 in Qatar.
His parents did lose 3 children because of Ada-Scid, and Abdul’s case is one of the most symbolic.
Just after his birth doctors did diagnose his illness and succeeded to healt his pneumonia during his first days of life.
So Abdul’s parents and doctors who were follow him contacted the San Raffaele Telethon Institute for Gene Therapy (Hsr-Tiget), starting files to bring him in Italy.

The child arrived in Italy only 3 months old, and subject to gene therapy. Researchers did take stem cells from his bone marrow and then injected in it a heal ADA gene into the diseased cells’ chromosomes.
To create enoguh room to host the right stem cells in the bone marrow Abdul did get a little dose of “busulfano” (a chemotherapeutic drug) before the gene therapy. After injection of the stem cells, these went back to the bone marrow and began to produce blood cells containing the “normal” ADA enzyme.
Scientists showed that right ADA cells continue to procude healthy blood in Abdul and in the other children even after years.

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