Spatial and subcellular gene expression patterns in myogenic differentiation and muscle pathology

Thesis

Duchenne muscular dystrophy (DMD) is a debilitating muscle-wasting disorder caused by lack of dystrophin protein. Promising dystrophin-restoration therapies such as gene editing using the CRISPR-Cas9 system and antisense oligonucleotide-mediated exon skipping using peptide-phosphorodiamidate morpholino oligomers (PPMOs) are currently in development. The efficacy of these approaches is typically assessed in terms of the amount of dystrophin restored, while correct sarcolemmal localisation o...

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Authors: Chwalenia, KM

• DOI: 10.5287/ora-pvzzygmbn
• Type of award: DPhil
• Level of award: Doctoral
• Awarding institution: University of Oxford
• Language: English
• Keywords: dystrophin; neuromuscular disorders; exon-skipping therapy; antisense oligonucleotides; microRNA; Duchenne muscular dystrophy