Rovere-Querini Patrizia, Clementi Emilio, Brunelli Silvia
Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele University and Scientific Institute, 20132 Milano, Italy.
Scientific Institute IRCCS Eugenio Medea, 23842 Bosisio Parini, Italy; Unit of Clinical Pharmacology, Consiglio Nazionale delle Ricerche, Institute of Neuroscience, Department of Biomedical and Clinical Sciences L. Sacco, University Hospital "Luigi Sacco", Università di Milano, 20157 Milan, Italy.
Eur J Pharmacol. 2014 May 5;730:181-5. doi: 10.1016/j.ejphar.2013.11.006. Epub 2013 Nov 20.
Muscular dystrophies comprise an heterogeneous group of diseases characterised by primary wasting of skeletal muscle, in the most severe forms leading to progressive paralysis and death. Current therapies for these conditions are extremely limited and based on corticosteroids that bear significant side effects. Several studies have proposed possible alternative strategies, ranging from cell and gene therapy to more classical pharmacological approaches. Nitric oxide is a gaseous messenger involved in many mechanisms responsible for preserving muscle function and stimulating muscle repair. We herein review the most recent pre-clinical and clinical findings that open new prospective for the development of nitric oxide as a therapeutic tool for muscular dystrophies.
肌营养不良症是一组异质性疾病,其特征是骨骼肌原发性萎缩,最严重的形式会导致进行性瘫痪和死亡。目前针对这些病症的治疗方法极为有限,且基于有显著副作用的皮质类固醇。多项研究提出了可能的替代策略,从细胞和基因疗法到更为传统的药理学方法。一氧化氮是一种气体信使,参与许多维持肌肉功能和刺激肌肉修复的机制。我们在此回顾了最新的临床前和临床研究结果,这些结果为一氧化氮作为肌营养不良症治疗工具的开发开辟了新的前景。