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

被引：94

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

页码：1674 / 1678

页数：5

共 36 条

[1] CLONING AND EXPRESSION OF THE MOUSE PGK-1 GENE AND THE NUCLEOTIDE-SEQUENCE OF ITS PROMOTER
ADRA, CN
BOER, PH
MCBURNEY, MW
[J]. GENE, 1987, 60 (01) : 65 - 74
[2] TRANSFER OF A FOREIGN GENE INTO THE BRAIN USING ADENOVIRUS VECTORS
AKLI, S
CAILLAUD, C
VIGNE, E
STRATFORDPERRICAUDET, LD
POENARU, L
PERRICAUDET, M
KAHN, A
PESCHANSKI, MR
[J]. NATURE GENETICS, 1993, 3 (03) : 224 - 228
[3] MICROGLIA EMERGE FROM THE FOG
ALTMAN, J
[J]. TRENDS IN NEUROSCIENCES, 1994, 17 (02) : 47 - 49
[4] GENE-TRANSFER INTO MAMMALIAN CENTRAL-NERVOUS-SYSTEM USING HERPES-VIRUS VECTORS - EXTENDED EXPRESSION OF BACTERIAL LACZ IN NEURONS USING THE NEURON-SPECIFIC ENOLASE PROMOTER
ANDERSEN, JK
GARBER, DA
MEANEY, CA
BREAKEFIELD, XO
[J]. HUMAN GENE THERAPY, 1992, 3 (05) : 487 - 499
[5] THE LEUKODYSTROPHIES - A WINDOW TO MYELIN
AUBOURG, P
[J]. NATURE GENETICS, 1993, 5 (02) : 105 - 106
[6] PSEUDOINFANTILE REFSUMS DISEASE - CATALASE-DEFICIENT PEROXISOMAL PARTICLES WITH PARTIAL DEFICIENCY OF PLASMALOGEN SYNTHESIS AND OXIDATION OF FATTY-ACIDS
AUBOURG, P
KREMSER, K
ROLAND, MO
ROCCHICCIOLI, F
SINGH, I
[J]. PEDIATRIC RESEARCH, 1993, 34 (03) : 270 - 276
[7] A 2-YEAR TRIAL OF OLEIC AND ERUCIC ACIDS (LORENZO OIL) AS TREATMENT FOR ADRENOMYELONEUROPATHY
AUBOURG, P
ADAMSBAUM, C
LAVALLARDROUSSEAU, MC
ROCCHICCIOLI, F
CARTIER, N
JAMBAQUE, I
JAKOBEZAK, C
LEMAITRE, A
BOUREAU, F
WOLF, C
BOUGNERES, PF
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 1993, 329 (11) : 745 - 752
[8] REVERSAL OF EARLY NEUROLOGIC AND NEURORADIOLOGICAL MANIFESTATIONS OF X-LINKED ADRENOLEUKODYSTROPHY BY BONE-MARROW TRANSPLANTATION
AUBOURG, P
BLANCHE, S
JAMBAQUE, I
ROCCHICCIOLI, F
KALIFA, G
NAUDSAUDREAU, C
ROLLAND, MO
DEBRE, M
CHAUSSAIN, JL
GRISCELLI, C
FISCHER, A
BOUGNERES, PF
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 1990, 322 (26) : 1860 - 1866
[9] AUBOURG P, 1985, J LIPID RES, V26, P263
[10] DIRECT INVIVO GENE-TRANSFER TO EPENDYMAL CELLS IN THE CENTRAL-NERVOUS-SYSTEM USING RECOMBINANT ADENOVIRUS VECTORS
BAJOCCHI, G
FELDMAN, SH
CRYSTAL, RG
MASTRANGELI, A
[J]. NATURE GENETICS, 1993, 3 (03) : 229 - 234

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