2012
DOI: 10.1016/j.nmd.2011.04.012 View full text |Buy / Rent full text
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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. Neither an effective treatment nor a cure is available at the present time. The mdx mouse has become the standard animal model for pre-clinical evaluation of potential therapeutic treatments. Recent years have seen a rapid incre… Show more

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“…The progression of pathology in the mdx mouse is influenced by growth (Grounds, 2008) and may be divided into three main phases: (1) the pre-weaning phase (0-3 weeks of age), which is strongly influenced by growth and corresponds roughly to the first 6 months of human patients, (2) the post-weaning phase, with an acute onset of pathology around 3 weeks, followed at about 8 weeks by (3) the adult phase with a reduced low level of chronic damage that persists throughout life (Willmann et al, 2011). An important degeneration and regeneration of muscle fibers is observed at a young age of mdx-mouse (2 to 4 weeks) which results in the muscle morphological changes such as centralized nuclei and heterogeneity of fiber size.…”
Section: Pathogenesis In the MDX Mousementioning
“…Many factors need to be considered. This protective effect will depend not only on the amount of dystrophin protein within an individual myofibers, but also on the extent of distribution within all myofibers, the size of the nuclear domain (how far dystrophin protein extends along the sarcolema from the myonuclei where the mRNA is generated) and on when during development the dystrophin must be produced (Willmann et al, 2011). This situation was considered by Chamberlain (1997) who concluded from analysis of mosaic transgenic mice and viral vector delivery with suboptimal doses into mdx mice, that >50% of myofibers need to express dystrophin, and that these must accumulate approximately 20% of wild-type levels of dystrophin for a significant correction of the muscle pathology in mice.…”
Section: Pre-clinical Therapeutic Studies Using Animal Modelsmentioning
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“…It has recently been proposed that a set of standard operating procedures be established for evaluating pre-clinical testing data in mdx mice (Grounds et al 2008;Nagaraju & Willmann 2009;Spurney et al 2009;Willmann et al 2011). Through the universal adoption of standardized laboratory assays, the results of multiple pre-clinical trials performed in independent laboratories could be evaulated.…”
Section: Current Progress and Evaluationmentioning