Small Gene Therapy Trial Success for Rare Degenerative Brain Disease – MedPage Today
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Small Gene Therapy Trial Success for Rare Degenerative Brain Disease
MedPage Today Gene therapy using autologous stem cell transplantation may be a safe and effective alternative to allogeneic transplant for treating cerebral adrenoleukodystrophy, researchers reported. In an open-label phase II/III safety and efficacy study, 15 of 17 … |
