International gene therapy trial for 'bubble boy' disease shows promising early results




Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome, a fatal genetic immunodeficiency also known as "bubble boy" disease, who were treated in an international clinical study of a new form of gene therapy. The mechanism that delivered the gene therapy is designed to prevent the leukemia that arose a decade ago in a similar trial in Europe, when one-quarter of boys treated developed the blood cancer.

Fuente : http://www.eurekalert.org/pub_releases/2013-12/dci...

Sábado, 7 de Diciembre 2013
Jueves, 1 de Enero 1970
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