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…