Peroxisome Proliferator Activated Receptor δ (PPARδ) Agonist But Not PPARα Corrects Carnitine Palmitoyl Transferase 2 Deficiency in Human Muscle Cells - Inserm - Institut national de la santé et de la recherche médicale Accéder directement au contenu
Article Dans Une Revue Journal of Clinical Endocrinology and Metabolism Année : 2005

Peroxisome Proliferator Activated Receptor δ (PPARδ) Agonist But Not PPARα Corrects Carnitine Palmitoyl Transferase 2 Deficiency in Human Muscle Cells

Résumé

Type 2 carnitine palmitoyl transferase (CPT2) is involved in the transfer of long-chain fatty acid into the mitochondria. CPT2-deficient patients carry gene mutations associated with different clinical presentations, correlating with various levels of fatty acid oxidation (FAO) and residual CPT2 enzyme activity. We tested the hypothesis that pharmacological stimulation of peroxisome proliferator-activated receptors (PPAR) can stimulate FAO in CPT2-deficient muscle cells. Accordingly, we show that a 48-h treatment of CPT2-deficient myoblasts by bezafibrate restored FAO in patient cells. Specific agonists of PPARdelta (GWdelta 0742), and, to a lower extent, PPARalpha (GWalpha 7647) also stimulated FAO in control myoblasts. However, when tested in CPT2-deficient myoblasts, only the delta-agonist was able to restore FAO, whereas the alpha-agonist had no effect. GWdelta 0742 increased CPT2 mRNA levels, whereas no change in CPT2 transcripts was found in response to GWalpha 7647. Bezafibrate and GWdelta 0742 increased residual CPT2 activity and normalized long-chain acylcarnitine production by deficient cells. Finally, CPT1-B mRNA was also stimulated after PPAR agonist treatment, and this likely takes part in drug-induced increase of FAO in control muscle cells. In conclusion, this study clearly suggests that PPARs could be therapeutic targets for correction of inborn beta-oxidation defects in human muscle. Furthermore, these data also illustrate a selective control of beta-oxidation enzyme gene expression by PPARdelta, with no contribution of PPARalpha.
Fichier sous embargo
Fichier sous embargo
Date de visibilité indéterminée
Loading...

Dates et versions

inserm-02896295 , version 1 (10-07-2020)

Identifiants

Citer

Fatima Djouadi, Flore Aubey, Dimitri Schlemmer, Jean Bastin. Peroxisome Proliferator Activated Receptor δ (PPARδ) Agonist But Not PPARα Corrects Carnitine Palmitoyl Transferase 2 Deficiency in Human Muscle Cells. Journal of Clinical Endocrinology and Metabolism, 2005, 90 (3), pp.1791-1797. ⟨10.1210/jc.2004-1936⟩. ⟨inserm-02896295⟩

Collections

INSERM
36 Consultations
2 Téléchargements

Altmetric

Partager

Gmail Facebook X LinkedIn More