Literature DB >> 7878038

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

N Cartier1, J Lopez, P Moullier, F Rocchiccioli, M O Rolland, P Jorge, J Mosser, J L Mandel, P F Bougnères, O Danos.   

Abstract

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.

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Year:  1995        PMID： 7878038      PMCID： PMC42582          DOI： 10.1073/pnas.92.5.1674

Source DB:  PubMed          Journal:  Proc Natl Acad Sci U S A        ISSN： 0027-8424            Impact factor:   11.205


  33 in total

1.  Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges.

Authors:  O Danos; R C Mulligan
Journal:  Proc Natl Acad Sci U S A       Date:  1988-09       Impact factor: 11.205

2.  Development of retrovirus vectors useful for expressing genes in cultured murine embryonal cells and hematopoietic cells in vivo.

Authors:  B C Guild; M H Finer; D E Housman; R C Mulligan
Journal:  J Virol       Date:  1988-10       Impact factor: 5.103

3.  Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo.

Authors:  W F Hickey; H Kimura
Journal:  Science       Date:  1988-01-15       Impact factor: 47.728

4.  X-linked adrenoleukodystrophy: identification of the primary defect at the level of a deficient peroxisomal very long chain fatty acyl-CoA synthetase using a newly developed method for the isolation of peroxisomes from skin fibroblasts.

Authors:  R J Wanders; C W van Roermund; M J van Wijland; R B Schutgens; A W Schram; J M Tager; H van den Bosch; C Schalkwijk
Journal:  J Inherit Metab Dis       Date:  1988       Impact factor: 4.982

5.  Recombinant retroviruses encoding simian virus 40 large T antigen and polyomavirus large and middle T antigens.

Authors:  P S Jat; C L Cepko; R C Mulligan; P A Sharp
Journal:  Mol Cell Biol       Date:  1986-04       Impact factor: 4.272

6.  Peroxisomal lignoceroyl-CoA ligase deficiency in childhood adrenoleukodystrophy and adrenomyeloneuropathy.

Authors:  O Lazo; M Contreras; M Hashmi; W Stanley; C Irazu; I Singh
Journal:  Proc Natl Acad Sci U S A       Date:  1988-10       Impact factor: 11.205

7.  Lignoceric acid is oxidized in the peroxisome: implications for the Zellweger cerebro-hepato-renal syndrome and adrenoleukodystrophy.

Authors:  I Singh; A E Moser; S Goldfischer; H W Moser
Journal:  Proc Natl Acad Sci U S A       Date:  1984-07       Impact factor: 11.205

8.  Lignoceroyl-CoASH ligase: enzyme defect in fatty acid beta-oxidation system in X-linked childhood adrenoleukodystrophy.

Authors:  M Hashmi; W Stanley; I Singh
Journal:  FEBS Lett       Date:  1986-02-17       Impact factor: 4.124

9.  Cloning and expression of the mouse pgk-1 gene and the nucleotide sequence of its promoter.

Authors:  C N Adra; P H Boer; M W McBurney
Journal:  Gene       Date:  1987       Impact factor: 3.688

10.  The gene responsible for adrenoleukodystrophy encodes a peroxisomal membrane protein.

Authors:  J Mosser; Y Lutz; M E Stoeckel; C O Sarde; C Kretz; A M Douar; J Lopez; P Aubourg; J L Mandel
Journal:  Hum Mol Genet       Date:  1994-02       Impact factor: 6.150
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  16 in total

Review 1.  X linked adrenoleukodystrophy: clinical presentation, diagnosis, and therapy.

Authors:  B M van Geel; J Assies; R J Wanders; P G Barth
Journal:  J Neurol Neurosurg Psychiatry       Date:  1997-07       Impact factor: 10.154

2.  ABCD1 deletion-induced mitochondrial dysfunction is corrected by SAHA: implication for adrenoleukodystrophy.

Authors:  Mauhamad Baarine; Craig Beeson; Avtar Singh; Inderjit Singh
Journal:  J Neurochem       Date:  2015-01-13       Impact factor: 5.372

Review 3.  X-linked adrenoleukodystrophy: genes, mutations, and phenotypes.

Authors:  K D Smith; S Kemp; L T Braiterman; J F Lu; H M Wei; M Geraghty; G Stetten; J S Bergin; J Pevsner; P A Watkins
Journal:  Neurochem Res       Date:  1999-04       Impact factor: 3.996

4.  A mouse model for X-linked adrenoleukodystrophy.

Authors:  J F Lu; A M Lawler; P A Watkins; J M Powers; A B Moser; H W Moser; K D Smith
Journal:  Proc Natl Acad Sci U S A       Date:  1997-08-19       Impact factor: 11.205

Review 5.  The Landscape of Hematopoietic Stem Cell Transplant and Gene Therapy for X-Linked Adrenoleukodystrophy.

Authors:  Eric J Mallack; Bela Turk; Helena Yan; Florian S Eichler
Journal:  Curr Treat Options Neurol       Date:  2019-11-25       Impact factor: 3.598

6.  Mutational and protein analysis of patients and heterozygous women with X-linked adrenoleukodystrophy.

Authors:  V Feigenbaum; G Lombard-Platet; S Guidoux; C O Sarde; J L Mandel; P Aubourg
Journal:  Am J Hum Genet       Date:  1996-06       Impact factor: 11.025

Review 7.  Biochemistry of peroxisomes in health and disease.

Authors:  I Singh
Journal:  Mol Cell Biochem       Date:  1997-02       Impact factor: 3.396

8.  A close relative of the adrenoleukodystrophy (ALD) gene codes for a peroxisomal protein with a specific expression pattern.

Authors:  G Lombard-Platet; S Savary; C O Sarde; J L Mandel; G Chimini
Journal:  Proc Natl Acad Sci U S A       Date:  1996-02-06       Impact factor: 11.205

9.  Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein.

Authors:  Muriel Asheuer; Françoise Pflumio; Sonia Benhamida; Anne Dubart-Kupperschmitt; Françoise Fouquet; Yoshinori Imai; Patrick Aubourg; Nathalie Cartier
Journal:  Proc Natl Acad Sci U S A       Date:  2004-02-27       Impact factor: 11.205

10.  Peroxisomal Acyl-CoA synthetase activity is essential for seedling development in Arabidopsis thaliana.

Authors:  Martin Fulda; Judy Schnurr; Amine Abbadi; Ernst Heinz; John Browse
Journal:  Plant Cell       Date:  2004-01-23       Impact factor: 11.277
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