Clinical benefit of lentiviral gene therapy in two patients with a rare neurologic disease
X-linked adrenoleukodystrophy (ALD) is a rare neurologic disease caused by a defect in a gene required for normal ABCD1 transporter function. The lack of this function leads to progressive demyelination, severe neurologic disease and death in males, often in childhood. ALD disease progression can be controlled by allogeneic hematopoietic cell transplantation (HCT) in those patients …