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filingDate 1999-09-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2002-10-01-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-6458838-B1
titleOfInvention Adrenoleukodystrophy treatments
abstract 4-phenylbutyrate treatment of cells from both X-ALD patients and X-ALD knockout mice results in decreased levels of and increased beta-oxidation of very-long-chain fatty acids; increased expression of the peroxisomal protein ALDRP; and induction of peroxisome proliferation. ALDP and ALDRP are functionally related, and ALDRP cDNA can complement X-ALD fibroblasts. Dietary 4-phenylbutyrate treatment is effective, producing a substantial reduction of very-long-chain fatty acid levels in the brain and adrenal glands of X-ALD mice.
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