Small molecules which improve pathogenesis of myotonic dystrophy type 1

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Small molecules which improve pathogenesis of myotonic dystrophy type 1

Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy in adults for which there is currently no treatment. The pathogenesis of this autosomal dominant disorder is associated with the expansion of CTG repeats in the 3′-UTR of the DMPK gene. DMPK transcripts with expanded CUG repeats (...

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Bibliographic Details
Main Authors:	López-Morató, Marta, Brook, J. David, Wojciechowska, Marzena
Format:	Article
Published:	Frontiers Media 2018
Subjects:	myotonic dystrophy type 1 myotonic dystrophy type 1 pathogenesis sequestration of muscleblind-like 1 antisense oligonucleotides aberrant alternative splicing small molecule compounds
Online Access:	https://eprints.nottingham.ac.uk/52545/

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