Gene redundancy and pharmacological gene therapy: Implications for X-linked adrenoleukodystrophy
β Scribed by Kemp, Stephan; Wei, He-Ming; Lu, Jyh-Feng; Braiterman, Lelita T; McGuinness, Martina C.; Moser, Ann B.; Watkins, Paul A.; Smith, Kirby D.
- Book ID
- 109747539
- Publisher
- Nature Publishing Group
- Year
- 1998
- Tongue
- English
- Weight
- 288 KB
- Volume
- 4
- Category
- Article
- ISSN
- 1078-8956
- DOI
- 10.1038/3242
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In its severe form, X-linked adrenoleukodystrophy (ALD) is a lethal neurologic disease of children, characterized by progressive cerebral demyelination and adrenal insufficiency. Associated with a biochemical defect of peroxisomal β€-oxidation, very long-chain fatty acids (VLCFA) build up in tissues