Early gene therapy results in Wiskott-Aldrich syndrome promising

Researchers reported promising preliminary outcomes for the first four children enrolled in a US gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder. All four have improved between nine and 24 months following treatment. Since undergoing treatment, none have experienced bleeding events or severe WAS-related infections. All four have experienced improvements in immunologic symptoms and variable improvements in platelet count.

from Today's Healthcare News -- ScienceDaily http://ift.tt/1NQ80iQ