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

被引：39

作者：

Flavigny, E ^{[1
]}

Sanhaj, A ^{[1
]}

Aubourg, P ^{[1
]}

Cartier, N ^{[1
]}

机构：

[1] Hop St Vincent de Paul, INSERM, U342, F-75014 Paris, France

来源：

FEBS LETTERS | 1999年 / 448卷 / 2-3期

关键词：

adrenoleukodystrophy; ATP-binding cassette transporter; peroxisome;

D O I：

10.1016/S0014-5793(99)00379-8

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

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. (C) 1999 Federation of European Biochemical Societies.

引用

页码：261 / 264

页数：4

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