Gene therapy appeared safe and effective for children with Wiskott-Aldrich syndrome without a suitable donor for hematopoietic stem cell transplantation, according to a study published in Nature Medicine.
“This work shows the potential for gene therapy to act as a safe alternative [for patients with Wiskott-Aldrich syndrome (WAS)] when transplant is a less favorable option,” Adrian J. Thrasher, MBBS, PhD, professor in pediatric immunology and Wellcome Trust principal research fellow at the University College London Great Ormond Street Institute of Child Health, said in a press