Scientists develop gene therapy for rare childhood disorder

12/6/2010 | Bloomberg Businessweek

German researchers said they were able to boost platelet counts and bolster some immune system cells in pediatric patients with Wiskott-Aldrich syndrome, a rare genetic disorder characterized by prolonged hemorrhage among children, using a gene therapy. The therapy involves the use of a viral vector to insert a healthy version of a faulty gene that causes the syndrome. However, one of the 10 children who received the treatment is undergoing chemotherapy after developing acute T-cell leukemia, which possibly was caused by the use of the viral vector.

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