Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy.

Adrenoleukodystrophy (ALD) is caused by mutations within the X-linked gene, resulting in the inability to transport acylated very long chain fatty acids (VLCFAs) into the peroxisome for degradation. VLCFAs subsequently accumulate in tissues, including the central nervous system. Up to 40% of boys develop a severe progressive demyelinating form of ALD, cerebral ALD, resulting in regions of demyelination observed on brain magnetic resonance imaging that are associated with a "garland ring" of gadolinium contrast enhancement.