Adrenoleukodystrophy

A Basis to Study ALD Gene Transfer in Stem Cells Is Reported

Published in Gene Therapy Weekly, April 3rd, 1995

Researchers from France stated that their study results offer a basis for in vitro study of the adrenoleukodystrophy (ALD) gene transfer and expression in hematopoietic stem cells.

Nathalie Cartier, Institut National de la Sante et de la Recherche Medicale, Paris, France, and colleagues noted that ADL is a lethal demyelinating brain disease ("Retroviral-Mediated Gene Transfer Corrects Very-Long-Chain Fatty Acid Metabolism in Adrenoleukodystrophy Fibroblasts," Proceedings of the National Academy of Sciences, February 1995;92:1674-1678).

ALD is caused by mutations of a gene encoding an ATP-binding transporter, which is called ALDP and localized in the...

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Source: Gene Therapy Weekly (1995-04-03)

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