Journal
NEUROBIOLOGY OF DISEASE
Volume 31, Issue 1, Pages 1-19Publisher
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.nbd.2008.03.008
Keywords
mdx mouse; muscular dystrophy; standard operating procedures; biological variation; muscle function; pre-clinical trials
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Funding
- NICHD NIH HHS [U54 HD053177, U54 HD053177-03S1] Funding Source: Medline
- Telethon [GGP05130] Funding Source: Medline
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This review discusses various issues to consider when developing standard operating procedures for preclinical 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. (C) 2008 Elsevier Inc. All rights reserved.
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