Retroviral‐mediated adrenoleukodystrophy‐related gene transfer corrects very long chain fatty acid metabolism in adrenoleukodystrophy fibroblasts: implications for therapy
Copyright policy )Retroviral‐mediated adrenoleukodystrophy‐related gene transfer corrects very long chain fatty acid metabolism in adrenoleukodystrophy fibroblasts: implications for therapy
X‐linked adrenoleukodystrophy is a demyelinating disorder of the central nervous system with an impaired very long chain fatty acid metabolism. The adrenoleukodystrophy gene encodes a peroxisomal membrane protein that is part of a family of related ATP‐binding transporters including the adrenoleukodystrophy‐related protein. The adrenoleukodystrophy protein and adrenoleukodystrophy‐related protein show 66% identity and have a mirror expression in most mouse tissues. We show that retroviral‐mediated adrenoleukodystrophy‐related gene transfer corrects very long chain fatty acid accumulation in adrenoleukodystrophy fibroblasts, irrespective of the presence or absence of adrenoleukodystrophy protein. Pharmacological approaches aiming at overexpressing the adrenoleukodystrophy‐related gene in the central nervous system of adrenoleukodystrophy patients might thus offer new therapeutic leads.
Fatty Acids, Gene Transfer Techniques, Peroxisome, Fibroblasts, Immunohistochemistry, Mice, Retroviridae, Transduction, Genetic, ATP-binding cassette transporter, Animals, Humans, Adrenoleukodystrophy, Cells, Cultured
Fatty Acids, Gene Transfer Techniques, Peroxisome, Fibroblasts, Immunohistochemistry, Mice, Retroviridae, Transduction, Genetic, ATP-binding cassette transporter, Animals, Humans, Adrenoleukodystrophy, Cells, Cultured
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