Early gene therapy results in Wiskott-Aldrich syndrome promising

(Dana-Farber Cancer Institute) 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, at the annual meeting of the American Society of Hematology. 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 impro...