Spurney Christopher F, Gordish-Dressman Heather, Guerron Alfredo D, Sali Arpana, Pandey Gouri S, Rawat Rashmi, Van Der Meulen Jack H, Cha Hee-Jae, Pistilli Emidio E, Partridge Terence A, Hoffman Eric P, Nagaraju Kanneboyina
Research Center for Genetic Medicine, Children's National Medical Center, 111 Michigan Avenue NW, Washington, DC 20010, USA.
Muscle Nerve. 2009 May;39(5):591-602. doi: 10.1002/mus.21211.
The availability of animal models for Duchenne muscular dystrophy has led to extensive preclinical research on potential therapeutics. Few studies have focused on reliability and sensitivity of endpoints for mdx mouse drug trials. Therefore, we sought to compare a wide variety of reported and novel endpoint measures in exercised mdx and normal control mice at 10, 20, and 40 weeks of age. Statistical analysis as well as power calculations for expected effect sizes in mdx preclinical drug trials across different ages showed that body weight, normalized grip strength, horizontal activity, rest time, cardiac function measurements, blood pressure, total central/peripheral nuclei per fiber, and serum creatine kinase are the most effective measurements for detecting drug-induced changes. These data provide an experimental basis upon which standardization of preclinical drug testing can be developed. Muscle Nerve, 2008.
杜兴氏肌肉营养不良症动物模型的可用性推动了针对潜在疗法的广泛临床前研究。很少有研究关注mdx小鼠药物试验终点的可靠性和敏感性。因此，我们试图比较10周、20周和40周龄的运动型mdx小鼠和正常对照小鼠中各种已报道的和新的终点测量方法。对不同年龄的mdx临床前药物试验中预期效应大小的统计分析以及功效计算表明，体重、标准化握力、水平活动、休息时间、心脏功能测量、血压、每根纤维的中央/外周核总数以及血清肌酸激酶是检测药物诱导变化的最有效测量方法。这些数据为制定临床前药物测试标准化提供了实验依据。《肌肉与神经》，2008年。
