RETROVIRAL-MEDIATED GENE-TRANSFER CORRECTS VERY-LONG-CHAIN FATTY-ACID METABOLISM IN ADRENOLEUKODYSTROPHY FIBROBLASTS

被引：95
作者：
CARTIER, N
LOPEZ, J
MOULLIER, P
ROCCHICCIOLI, F
ROLLAND, MO
JORGE, P
MOSSER, J
MANDEL, JL
BOUGNERES, PF
DANOS, O
AUBOURG, P
机构：
[1] INST PASTEUR,RETROVIRUS & TRANSFERT GENET LAB,CNRS,URA 1157,F-75724 PARIS 15,FRANCE
[2] HOP DEBROUSSE,DEPT BIOCHEM,F-69322 LYON 05,FRANCE
[3] FAC MED STRASBOURG,GENET MOLEC EUCARYOTES LAB,INSERM,U184,F-67085 STRASBOURG,FRANCE
[4] CHRU STRASBOURG,F-67085 STRASBOURG,FRANCE
来源：
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA | 1995年 / 92卷 / 05期
关键词：
PEROXISOMES; RETROVIRAL VECTOR; GENE THERAPY;
D O I：
10.1073/pnas.92.5.1674
中图分类号：
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号：
07 ; 0710 ; 09 ;
摘要：
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.
引用
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页码：1674 / 1678
页数：5
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