Investigating genome engineering of myotonic dystrophy cells and small molecules as potential therapeutic agents

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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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Bibliographic Details
Main Author:	López Morató, Marta
Format:	Thesis (University of Nottingham only)
Language:	English
Published:	2018
Subjects:	PhD Myotonic dystrophy CRISPR Therapy Small molecules DM1 DM2
Online Access:	https://eprints.nottingham.ac.uk/55585/

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