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.

View Full Article in:

Bloomberg Businessweek

Published in Brief:

SmartBrief Job Listings for Health Care

Job Title Company Location
Regulatory Counsel
Food and Drug Administration
Silver Spring, MD
Pharmacy Care Manager
National Association of Chain Drug Stores
Arlington, VA
Vice President, Commercial Markets
Meridian Health
Neptune, NJ
Health Plan CEO – Oregon
Trillium Community Health Plan
Eugene, OR
Director of Operations - GBS
Mayo Clinic
Rochester, MN