A U.S. gene therapy trial for a life-threatening genetic blood and immune disorder, Wiskott-Aldrich syndrome, is producing promising preliminary results, according to study principal investigator, Sung-Yun Pai, who is a pediatric hematologist-oncologist from Dana-Farber/Boston Children's Cancer and Blood Disorders Center. However, the study will have to monitor the trial patients, four children with WAS, for 15 years before conclusions can be drawn. At present, the only treatment is a hematopoietic stem cell transplant from a compatible donor but the trial is experimenting with a self-inactivating lentivirus vector that has been engineered to avoid triggering the development of leukemia.
A US gene therapy trial for a life-threatening genetic blood and immune disorder, Wiskott-Aldrich syndrome, is producing promising preliminary results, according to study principal investigator, Sung-Yun Pai, who is a pediatric hematologist-oncologist from Dana-Farber/Boston Children's Cancer and Blood Disorders Center. However, the study will have to monitor the trial patients, four children with WAS, for 15 years before conclusions can be drawn. At present, the only treatment is a hematopoietic stem cell transplant from a compatible donor but the trial is experimenting with a self-inactivating lentivirus vector that has been engineered to avoid triggering the development of leukemia.