The potential therapeutic effect of spermidine supplementation on the mdx mouse phenotype

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Giourmas, Nicholas (2021) The potential therapeutic effect of spermidine supplementation on the mdx mouse phenotype. Research Master thesis, Victoria University.

Abstract

Duchenne Muscular Dystrophy (DMD) is one of the most severe forms of inheritable muscular dystrophies that affects 1 in every 5,000 boys. DMD is caused by a genetic mutation on the X chromosome, which results in the loss of the full-length protein, dystrophin. Dystrophin plays a stabilising role by connecting the cytoskeleton of muscle fibres to the extracellular matrix, with the absence of dystrophin directly correlating with the severity of DMD. It has been previously shown that the drug, rapamycin, improves dystrophic muscle function, in part, through the upregulation of the process known as autophagy. This autophagic process plays a role in degrading/removing damaged molecules, including lipids and proteins. Long term use of rapamycin, however, may result in toxic side effects that makes its use as a DMD treatment limited. Because of this, finding a non-toxic inducer of autophagy may be beneficial in treating DMD. One molecule that is known to activate autophagy in a range of tissues, including skeletal muscle, is the polyamine spermidine.

Additional Information

Master of Applied Research

Item type Thesis (Research Master thesis)
URI https://vuir.vu.edu.au/id/eprint/43403
Subjects Current > FOR (2020) Classification > 3101 Biochemistry and cell biology
Current > Division/Research > Institute for Health and Sport
Keywords Duchenne Muscular Dystrophy, DMD, polyamine spermidine, skeletal muscle, autophagy, mice
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