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J Neuroimmunol. 2007 Jan;182(1-2):5-12. Epub 2006 Nov 27.

Immunopathogenesis of adrenoleukodystrophy: current understanding.

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Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Bunting Blaustein Cancer Research Building, Rm 255 1650 Orleans Street Baltimore, MD 21231, United States.


Adrenoleukodystrophy is a neurometabolic disease with a decreased ability to degrade very long chain fatty acids (VLCFA) and significant phenotypic variation. Unlike most neurometabolic diseases, the success of hematopoietic stem cell transplantation (HSCT) is based on acquiring a new immune system rather than enzyme replacement. VLCFA accumulation appears necessary but not sufficient for pathogenesis. Evidence for the involvement of different components of the immune system in the pathogenesis of the cerebral lesions (cellular, cytokines, humoral, and complement) is reviewed, along with both HSCT and non-HSCT immunologic approaches to treatment and future directions.

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