Enhancing translation: Guidelines for standard pre-clinical experiments in mdx mice

Raffaella Willmann, Annamaria De Luca, Michael Benatar, Miranda Grounds, Judith Dubach, Jean Marc Raymackers, Kanneboyina Nagaraju

Research output: Contribution to journalArticle

48 Scopus citations

Abstract

Duchenne Muscular Dystrophy is an X-linked disorder that affects boys and leads to muscle wasting and death due to cardiac involvement and respiratory complications. The cause is the absence of dystrophin, a large structural protein indispensable for muscle cell function and viability. The mdx mouse has become the standard animal model for pre-clinical evaluation of potential therapeutic treatments. Recent years have seen a rapid increase in the number of experimental compounds being evaluated in the mdx mouse. There is, however, much variability in the design of these pre-clinical experimental studies. This has made it difficult to interpret and compare published data from different laboratories and to evaluate the potential of a treatment for application to patients. The authors therefore propose the introduction of a standard study design for the mdx mouse model. Several aspects, including animal care, sampling times and choice of tissues, as well as recommended endpoints and methodologies are addressed and, for each aspect, a standard procedure is proposed. Testing of all new molecules/drugs using a widely accepted and agreed upon standard experimental protocol would greatly improve the power of pre-clinical experimentations and help identifying promising therapies for the translation into clinical trials for boys with Duchenne Muscular Dystrophy.

Original languageEnglish (US)
Pages (from-to)43-49
Number of pages7
JournalNeuromuscular Disorders
Volume22
Issue number1
DOIs
StatePublished - Jan 2012

Keywords

  • DMD
  • Mdx
  • Methods
  • Pre-clinical experiments
  • Standard operating procedures

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Genetics(clinical)
  • Neurology

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    Willmann, R., De Luca, A., Benatar, M., Grounds, M., Dubach, J., Raymackers, J. M., & Nagaraju, K. (2012). Enhancing translation: Guidelines for standard pre-clinical experiments in mdx mice. Neuromuscular Disorders, 22(1), 43-49. https://doi.org/10.1016/j.nmd.2011.04.012