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. 1995 Feb 28;92(5):1674–1678. doi: 10.1073/pnas.92.5.1674

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

N Cartier 1, J Lopez 1, P Moullier 1, F Rocchiccioli 1, M O Rolland 1, P Jorge 1, J Mosser 1, J L Mandel 1, P F Bougnères 1, O Danos 1, et al.
PMCID: PMC42582  PMID: 7878038

Abstract

Adrenoleukodystrophy (ALD), a lethal demyelinating disease of the brain, is caused by mutations of a gene encoding an ATP-binding transporter, called ALDP, localized in the peroxisomal membrane. It is associated with a defective oxidation of very-long-chain fatty acids, leading to their accumulation in many tissues. This study reports that the retroviral-mediated transfer of the ALD cDNA restored very-long-chain fatty acid oxidation in ALD fibroblasts in vitro following abundant expression and appropriate targeting of the vector-encoded ALDP in peroxisomes. The same method may be used in hematopoietic cells as a further step of a gene therapy approach of ALD.

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