Retroviral-mediated adrenoleukodystrophy-related gene transfer corrects very long chain fatty acid metabolism in adrenoleukodystrophy fibroblasts: implications for therapy

FEBS Lett. 1999 Apr 9;448(2-3):261-4. doi: 10.1016/s0014-5793(99)00379-8.

Abstract

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adrenoleukodystrophy / genetics*
  • Adrenoleukodystrophy / metabolism
  • Adrenoleukodystrophy / therapy*
  • Animals
  • Cells, Cultured
  • Fatty Acids / metabolism
  • Fibroblasts / cytology
  • Fibroblasts / metabolism*
  • Gene Transfer Techniques*
  • Humans
  • Immunohistochemistry
  • Mice
  • Retroviridae*
  • Transduction, Genetic

Substances

  • Fatty Acids