Haidet Amanda M, Rizo Liza, Handy Chalonda, Umapathi Priya, Eagle Amy, Shilling Chris, Boue Daniel, Martin Paul T, Sahenk Zarife, Mendell Jerry R, Kaspar Brian K
The Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Proc Natl Acad Sci U S A. 2008 Mar 18;105(11):4318-22. doi: 10.1073/pnas.0709144105. Epub 2008 Mar 11.
Increasing the size and strength of muscles represents a promising therapeutic strategy for musculoskeletal disorders, and interest has focused on myostatin, a negative regulator of muscle growth. Various myostatin inhibitor approaches have been identified and tested in models of muscle disease with varying efficacies, depending on the age at which myostatin inhibition occurs. Here, we describe a one-time gene administration of myostatin-inhibitor-proteins to enhance muscle mass and strength in normal and dystrophic mouse models for >2 years, even when delivered in aged animals. These results demonstrate a promising therapeutic strategy that warrants consideration for clinical trials in human muscle diseases.
增加肌肉的大小和力量是治疗肌肉骨骼疾病的一种有前景的策略，人们的兴趣集中在肌肉生长抑制素上，它是肌肉生长的负调节因子。已经确定了各种肌肉生长抑制素抑制剂方法，并在肌肉疾病模型中进行了测试，其疗效各不相同，这取决于抑制肌肉生长抑制素的年龄。在这里，我们描述了一次性给予肌肉生长抑制素抑制蛋白基因，以增强正常和营养不良小鼠模型中的肌肉质量和力量，持续超过2年，即使是在老年动物中给药也是如此。这些结果证明了一种有前景的治疗策略，值得在人类肌肉疾病的临床试验中加以考虑。
