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The World in Medicine
December 16, 2009

Gene Therapy and ALD

JAMA. 2009;302(23):2531. doi:10.1001/jama.2009.1841

An international team of researchers reports that gene therapy that addresses the genetic defect that underlies the brain demyelination disorder X-linked adrenoleukodystrophy (ALD) succeeded in halting progression of the disease in 2 young boys (Cartier N et al. Science. 2009;326[5954]818-823).

ADL, which is caused by mutations in the gene that encodes the ALD protein, can be treated with hematopoietic stem cell transplantation, but the disorder is fatal when a suitable matched donor is not available. The new approach involves removing blood stem cells from the patient and using a lentiviral vector derived from a disabled version of HIV to introduce a working copy of the ALD gene into these cells. The patient then receives an infusion of the modified cells after undergoing myeloablative treatment to destroy the bone marrow.