Investigating genome engineering of myotonic dystrophy cells and small molecules as potential therapeutic agents
Myotonic dystrophy (DM) is a dominantly inherited multisystem disease. There are 2 types of DM: DM1, caused by a CTG repeat expansion in the 3’-UTR of the DMPK gene, and DM2, caused by a CCTG repeat expansion in intron 1 of the CNBP gene. In both types of DM the transcribed RNA, containing the repea...
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| Format: | Thesis (University of Nottingham only) |
| Language: | English |
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2018
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| Online Access: | https://eprints.nottingham.ac.uk/55585/ |