Institute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Inherited and Acquired Myopathies Program, Australian Institute for Musculoskeletal Science (AIMSS), St Albans, Victoria, Australia.
FASEB J. 2024 Jun 15;38(11):e23718. doi: 10.1096/fj.202400329R.
Female carriers of a Duchenne muscular dystrophy (DMD) gene mutation manifest exercise intolerance and metabolic anomalies that may be exacerbated following menopause due to the loss of estrogen, a known regulator of skeletal muscle function and metabolism. Here, we studied the impact of estrogen depletion (via ovariectomy) on exercise tolerance and muscle mitochondrial metabolism in female mdx mice and the potential of estrogen replacement therapy (using estradiol) to protect against functional and metabolic perturbations. We also investigated the effect of estrogen depletion, and replacement, on the skeletal muscle proteome through an untargeted proteomic approach with TMT-labelling. Our study confirms that loss of estrogen in female mdx mice reduces exercise capacity, tricarboxylic acid cycle intermediates, and citrate synthase activity but that these deficits are offset through estrogen replacement therapy. Furthermore, ovariectomy downregulated protein expression of RNA-binding motif factor 20 (Rbm20), a critical regulator of sarcomeric and muscle homeostasis gene splicing, which impacted pathways involving ribosomal and mitochondrial translation. Estrogen replacement modulated Rbm20 protein expression and promoted metabolic processes and the upregulation of proteins involved in mitochondrial dynamics and metabolism. Our data suggest that estrogen mitigates dystrophinopathic features in female mdx mice and that estrogen replacement may be a potential therapy for post-menopausal DMD carriers.
女性 Duchenne 肌营养不良症 (DMD) 基因突变携带者表现出运动不耐受和代谢异常,由于雌激素的丧失,这些异常可能在绝经后加剧,因为雌激素是骨骼肌功能和代谢的已知调节剂。在这里,我们研究了雌激素耗竭(通过卵巢切除术)对雌性 mdx 小鼠的运动耐受力和肌肉线粒体代谢的影响,以及雌激素替代疗法(使用雌二醇)对预防功能和代谢紊乱的潜在作用。我们还通过 TMT 标记的非靶向蛋白质组学方法研究了雌激素耗竭和替代对骨骼肌蛋白质组的影响。我们的研究证实,雌性 mdx 小鼠中雌激素的丧失会降低运动能力、三羧酸循环中间产物和柠檬酸合酶活性,但这些缺陷可以通过雌激素替代疗法得到弥补。此外,卵巢切除术下调了 RNA 结合基序因子 20 (Rbm20) 的蛋白表达,Rbm20 是肌节和肌肉稳态基因剪接的关键调节因子,这影响了涉及核糖体和线粒体翻译的途径。雌激素替代调节 Rbm20 蛋白表达,并促进代谢过程和线粒体动力学和代谢相关蛋白的上调。我们的数据表明,雌激素减轻了雌性 mdx 小鼠的肌营养不良蛋白病特征,雌激素替代可能是绝经后 DMD 携带者的一种潜在治疗方法。