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San Francisco Chronicle
International Gene Therapy Trial for 'Bubble Boy' Disease Shows Promising ...
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Eight of the nine boys registered to date in the new trial are alive and well, with functioning immune systems and free of infections associated with SCID-X1, between nine and 36 months following treatment, according to Sung-Yun Pai, MD, a pediatric hematologist-oncologist from Dana-Farber/Boston Children's Cancer and Blood Disorders Center. She presented the findings at the 55th annual meeting of the American Society of Hematology on behalf of the Transatlantic Gene Therapy Consortium (TAGTC). The investigators continue to monitor the children for signs of treatment-associated leukemia, which developed three to five years post-treatment in the prior trial. They point to surrogate biological markers that give them hope the viral vector used to deliver the new treatment is safe.