6533b873fe1ef96bd12d4b50
RESEARCH PRODUCT
Pharmacological Induction of Redundant Genes for a Therapy of X-ALD
Maurice BugautFabien GueugnonJohannes BergerStéphane FourcadeStéphane SavaryFrank RoelsThierry PineauPascal G.p. MartinFrançoise CadepondAngela NetikCatherine GondcailleMartine El EtrMarianne Depretersubject
Pristanic acidcongenital hereditary and neonatal diseases and abnormalitiesFenofibrateendocrine system diseasesPharmacological therapybiologyPharmacologymedicine.diseasePhenylbutyrateBiochemical phenotypechemistry.chemical_compoundchemistrymedicineABCD2biology.proteinAdrenoleukodystrophyGenemedicine.drugdescription
X-linked adrenoleukodystrophy (X-ALD) is a recessive neurologic disease with an incidence among males of 1/17 000. Since the identification of the X-ALD gene (ABCD1) ten years ago (Mosser et al 1993), no satisfactory therapy has been available. A close homologue (ABCD2) was then cloned and presented as a putative modifier gene that could account for some of the extreme phenotypic variability of X-ALD (Lombard-Platet et al 1996). The inducibility of Abcd2 by the hypolipidemic drug fenofibrate in the liver of rodents (Albet et al 1997), correlated to a partial normalisation of the biochemical phenotype of X-ALD (Netik et al 1999), opened up the way of a pharmacological therapy of X-ALD. The basis of such a therapy and the results obtained chiefly in rodents will be presented in this chapter.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2003-01-01 |