Towards developing standard operating procedures for pre-clinical testing in the mdx mouse model of Duchenne muscular dystrophy

Neurobiology of Disease

Hannah G. Radley¹  Kanneboyina Nagaraju²  Annamaria De Luca³  Miranda D. Grounds⁴  Gordon S. Lynch⁴ 

[1] Corresponding author. Fax: [phone].;Basic and Clinical Myology Laboratory, Department of Physiology, the University of Melbourne, Victoria, Australia;Research Centre for Genetic Medicine, Murine Drug Testing Facility, Children's National Medical Center, Washington, USA;School of Anatomy and Human Biology, the University of Western Australia, Perth, Western Australia, Australia;

关键词: Mdx mouse;    Muscular dystrophy;    Standard operating procedures;    Biological variation;    Muscle function;    Pre-clinical trials;   

DOI  :  

来源: DOAJ

【 摘 要 】

This review discusses various issues to consider when developing standard operating procedures for pre-clinical studies in the mdx mouse model of Duchenne muscular dystrophy (DMD). The review describes and evaluates a wide range of techniques used to measure parameters of muscle pathology in mdx mice and identifies some basic techniques that might comprise standardised approaches for evaluation. While the central aim is to provide a basis for the development of standardised procedures to evaluate efficacy of a drug or a therapeutic strategy, a further aim is to gain insight into pathophysiological mechanisms in order to identify other therapeutic targets. The desired outcome is to enable easier and more rigorous comparison of pre-clinical data from different laboratories around the world, in order to accelerate identification of the best pre-clinical therapies in the mdx mouse that will fast-track translation into effective clinical treatments for DMD.

【 授权许可】

Unknown