Nearing Marie, Novak James, Partridge Terence
Children's National Health System, Children's Research Institute, Center for Genetic Medicine Research, Washington DC, United States.
Center for Genetic Medicine Research, Children's National Health System, Washington DC, United States.
PLoS Curr. 2018 May 2;10:ecurrents.md.230ed3d6559b171e10279fc16e9ebef3. doi: 10.1371/currents.md.230ed3d6559b171e10279fc16e9ebef3.
Duchenne Muscular Dystrophy is a genetic disease that is caused by a deficiency of dystrophin protein. Both Duchenne Muscular Dystrophy patients and dystrophic mice suffer from intestinal dysfunction.
The present study arose from a chance observation of differences in fecal output of dystrophic vs. normal mice during 20-minutes of forced continuous treadmill exercise. Here, we report on the effects of exercise on fecal output in two different dystrophic mutants and their normal background control strains. All fecal materials evacuated during exercise were counted, dried and weighed.
Mice of both mutant dystrophic strains produced significantly more fecal material during the exercise bout than the relevant control strains.
We propose that exercise--induced Colo--Rectal Activation Phenotype test could be used as a simple, highly sensitive, non-invasive biomarker to determine efficacy of dystrophin replacement therapies.
杜氏肌营养不良症是一种由肌营养不良蛋白缺乏引起的遗传性疾病。杜氏肌营养不良症患者和患营养不良症的小鼠都存在肠道功能障碍。
本研究源于在20分钟的强制连续跑步机运动期间偶然观察到患营养不良症的小鼠与正常小鼠粪便排出量的差异。在此,我们报告运动对两种不同的营养不良突变体及其正常背景对照品系粪便排出量的影响。对运动期间排出的所有粪便进行计数、干燥和称重。
两种突变型营养不良品系的小鼠在运动期间产生的粪便量均显著多于相应的对照品系。
我们提出运动诱导的结肠 - 直肠激活表型测试可作为一种简单、高度敏感、非侵入性的生物标志物,用于确定肌营养不良蛋白替代疗法的疗效。