Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy.
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DOI: 10.1182/blood-2018-11-887240
Abstract: Adrenoleukodystrophy (ALD) is caused by mutations within the X-linked ABCD1 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… read more here.
Keywords: blood brain; successful donor; adrenoleukodystrophy; blood ... See more keywords