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Gene redundancy and pharmacological gene therapy: Implications for X-linked adrenoleukodystrophy
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Gene redundancy and pharmacological gene therapy: Implications for X-linked adrenoleukodystrophy
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Journal Article
Gene redundancy and pharmacological gene therapy: Implications for X-linked adrenoleukodystrophy
1998
Overview
As more functional redundancy in mammalian cells is discovered, enhanced expression of genes involved in alternative pathways may become an effective form of gene therapy. X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal disorder with impaired very-long-chain fatty acid metabolism. The X-ALD gene encodes a peroxisomal membrane protein (ALDP) that is part of a small family of related peroxisomal membrane proteins. We show that 4-phenylbutyrate treatment of cells from both X-ALD patients and X-ALD knockout mice results in decreased levels of and increased β-oxidation of very-long-chain fatty acids; increased expression of the peroxisomal protein ALDRP; and induction of peroxisome proliferation. We also demonstrate that ALDP and ALDRP are functionally related, by ALDRP cDNA complementation of X-ALD fibroblasts. Finally, we demonstrate the
in vivo
efficacy of dietary 4-phenylbutyrate treatment through its production of a substantial reduction of very-long-chain fatty acid levels in the brain and adrenal glands of X-ALD mice.
Publisher
Nature Publishing Group US
Subject
Adrenoleukodystrophy - genetics
/ Adrenoleukodystrophy - therapy
/ Animals
/ ATP Binding Cassette Transporter, Sub-Family D
/ ATP Binding Cassette Transporter, Sub-Family D, Member 1
/ ATP-Binding Cassette Transporters - genetics
/ Biomedical and Life Sciences
/ Humans
/ Mice
/ Microbodies - ultrastructure
/ Phenylbutyrates - pharmacology
