Hematopoietic Stem-Cell Gene Therapy Is Associated with Restored White Matter Microvascular Function in Cerebral Adrenoleukodystrophy

Investigators analyzed cerebral perfusion imaging in boys with cerebral adrenoleukodystrophy treated with autologous hematopoietic stem-cells transduced with the Lenti-D lentiviral vector that contained ABCD1 cDNA and patients treated with allogeneic hematopoietic stem cell transplantation.