Lenti‐D for adrenoleukodystrophy in boys

Adrenoleukodystrophy (ALD), otherwise known as Lorenzo’s oil disease, is a rare genetic disorderlinked to the X chromosome (i.e. mainly affecting boys and men, although female carriers can exhibitsymptoms). Mutations in a gene called ABCD1 initiate a set of problems which result in the destructionof the protective sheath around nerve cells, and lead to progressive neurologic deterioration. The formof the disease most commonly found in children is called cerebral adrenoleukodystrophy (CALD) andis the most severe; if untreated it progresses rapidly and causes brain damage and eventually death inmost patients.The only treatment currently available is allogeneic haematopoietic stem cell transplant (HCT). Theproposed treatment involves harvesting stem cells from the patient's bone marrow and inserting ahealthy version of the ABCD1 gene using Lenti‐D. Defective cells are destroyed with chemotherapy,and the healthy cells are grown in culture and re‐injected into the patient.