6533b7d7fe1ef96bd1267a14
RESEARCH PRODUCT
A novel cell model to study the function of the adrenoleukodystrophy-related protein
Catherine GondcailleStephan KempFabien GueugnonNatalia VolodinaRonald J.a. WandersAnabelle Sequeira-le GrandJaoued Et TaouilPetra A.w. MooijerTatiana LopezStéphane Savarysubject
Carcinoma Hepatocellularendocrine system diseasesRecombinant Fusion ProteinsBiophysicsGene redundancyATP-binding cassette transporterContext (language use)BiologyATP Binding Cassette Transporter Subfamily DProtein EngineeringTransfectionBiochemistryCell Line TumormedicineAnimalsAdrenoleukodystrophyMolecular BiologyGeneCell BiologyPeroxisomemedicine.diseaseFusion proteinRatsCell biologyDisease Models AnimalBiochemistryATP-Binding Cassette TransportersAdrenoleukodystrophyFunction (biology)description
X-linked adrenoleukodystrophy (X-ALD) is a neurodegenerative disorder due to mutations in the ABCD1 (ALD) gene. ALDRP, the closest homolog of ALDP, has been shown to have partial functional redundancy with ALDP and, when overexpressed, can compensate for the loss-of-function of ALDP. In order to characterize the function of ALDRP and to understand the phenomenon of gene redundancy, we have developed a novel system that allows the controlled expression of the ALDRP-EGFP fusion protein (normal or non-functional mutated ALDRP) using the Tet-On system in H4IIEC3 rat hepatoma cells. The generated stable cell lines express negligible levels of endogenous ALDRP and doxycycline dosage-dependent levels of normal or mutated ALDRP. Importantly, the ALDRP-EGFP protein is targeted correctly to peroxisome and is functional. The obtained cell lines will be an indispensable tool in our further studies aimed at the resolution of the function of ALDRP to characterize its potential substrates in a natural context.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2006-01-01 |