Tag Archive for 'Immunodeficiency'

ITALY – Stem Cells. New Method to Combat Immunodeficiencies in Children at San Raffaele in Milan.

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Stem Man
on 13 October 2011
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The Angel
Image by pianetatschai via Flickr

It was stunning to see them closed inside of those plastic bubbles, kept far from all external contact because their immune system does not react against any foreign antigens. Today scientists can say that ADA-SCID (adenosine deaminase deficiency), a serious combined immunodeficiency caused due to a lack of the adenosine deaminase enzyme, has been definitively defeated by gene therapy developed at San Raffaele of Milan.

The final study, which combined the conclusions of clinical studies, which began in 2000 on strategic therapies developed by the HSR-TIGET (San Raffaele Telethon Institute for Gene Therapy) group, led by Maria Grazia Roncarolo and Alessandro Aiuti, and was published in the New England Journal of Medicine.

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