Characterising and evaluating the efficacy of metabolic therapies for the treatment of Duchenne Muscular Dystrophy

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Timpani, Cara (2017) Characterising and evaluating the efficacy of metabolic therapies for the treatment of Duchenne Muscular Dystrophy. PhD thesis, Victoria University.

Abstract

Duchenne Muscular Dystrophy (DMD) is a fatal skeletal muscle wasting disease underpinned by extensive metabolic dysfunction. This culminates in reduced energy production which is detrimental to dystrophic muscle since buffering damage and stimulating repair are energy dependent processes. Current treatment options for DMD are limited and do not address this metabolic dysfunction despite the extensive role it plays in DMD pathogenesis and disease progression. Therefore, this thesis investigated the efficacy of two metabolic therapies, sodium nitrate and adenylosuccinic acid (ASA), to improve skeletal muscle metabolism and architecture in the well-established mdx mouse model of DMD, and in immortalised myoblasts derived from DMD patients.

Additional Information

Includes bibliographical references pp. 324-412

Item type Thesis (PhD thesis)
URI https://vuir.vu.edu.au/id/eprint/34336
Subjects Current > FOR Classification > 1101 Medical Biochemistry and Metabolomics
Current > FOR Classification > 1109 Neurosciences
Current > Division/Research > College of Health and Biomedicine
Keywords neuromuscular diseases, muscle wasting, DMD, metabolism, metabolic system, metabogenic therapy, ASA therapy, treatment, mice, mitochondrial, histopathology, nitrate supplementation, nitrite supplementation, supplements, adenylosuccinic acid
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