PPARδ modulation rescues mitochondrial fatty acid oxidation defects in the mdx model of muscular dystrophy
PPARδ modulation rescues mitochondrial fatty acid oxidation defects in the mdx model of muscular dystrophy
复制标题
DOI：
10.1016/j.mito.2018.02.006
复制
发表时间：
2019-05-01
期刊：
影响因子：
4.4
通讯作者：
Tozzo, Effie
中科院分区：
文献类型：
--
作者：
Bell, Eric L.;Shine, Robert W.;Tozzo, Effie
杜氏肌营养不良症（DMD）是一种隐性的、致命的x连锁疾病，其特征是由于缺乏肌营养不良蛋白而导致的进行性骨骼肌萎缩，肌营养不良蛋白是一种连接细胞内骨架和细胞外基质的必需蛋白质。本研究旨在对mdx小鼠和野生型对照小鼠的原代肌卫星细胞衍生成肌细胞的线粒体进行表征。与野生型衍生细胞相比，mdx衍生细胞线粒体生物能量减少，线粒体数量减少。在这里，我们证明了一种新的PPAR delta调节剂可以改善mdx小鼠的线粒体功能，这支持了调节PPAR delta可能对DMD患者的治疗有益。
Duchenne muscular dystrophy (DMD) is a recessive, fatal X-linked disease that is characterized by progressive skeletal muscle wasting due to the absence of dystrophin, which is an a essential protein that bridges the inner cytoskeleton and extra-cellular matrix. This study set out to characterize the mitochondria in primary muscle satellite cell derived myoblasts from mdx mice and wild type control mice. Compared to wild type derived cells the mdx derived cells have reduced mitochondrial bioenergetics and have fewer mitochondria. Here, we demonstrate that a novel PPAR delta modulator improves mitochondrial function in the mdx mice, which supports that modulating PPAR delta may be therapeutically beneficial in DMD patients.