Adrenoleukodystrophy

Gene Transfer Corrects Fatty Acid Metabolism Problems

Published in Gene Therapy Weekly, May 24th, 1999

Retroviral-mediated gene transfer corrected long chain fatty acid metabolism in adrenoleukodystrophy fibroblasts.

X-linked adrenoleukodystrophy is a demyelinating disorder of the central nervous system which impairs 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, noted researchers E. Flavigny and colleagues from the Hopital St. Vincent de Paul, France ("Retroviral-Mediated Adrenoleukodystrophy-Related Gene Transfer Corrects Very Long Chain Fatty Acid Metabolism in Adrenoleukodystrophy Fibroblasts: ...

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Source: Gene Therapy Weekly (1999-05-24)

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