Targeted inactivation of the X-linked ad
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Sonja Forss-Petter; Hauke Werner; Johannes Berger; Hans Lassmann; Brunhilde Molz
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Article
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1997
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John Wiley and Sons
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English
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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 โค-oxidation, very long-chain fatty acids (VLCFA) build up in tissues