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

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...

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Publicado no:Proc Natl Acad Sci U S A
Principais autores: Cartier, N, Lopez, J, Moullier, P, Rocchiccioli, F, Rolland, M O, Jorge, P, Mosser, J, Mandel, J L, Bougnères, P F, Danos, O
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 1995
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC42582/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/7878038/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.92.5.1674
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