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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Auteurs principaux:	Marta López-Morató, John David Brook, Marzena Wojciechowska
Format:	Artigo
Langue:	Inglês
Publié:	Frontiers Media S.A. 2018-05-01
Collection:	Frontiers in Neurology
Sujets:	myotonic dystrophy type 1 myotonic dystrophy type 1 pathogenesis sequestration of muscleblind-like 1 antisense oligonucleotides aberrant alternative splicing small molecule compounds
Accès en ligne:	https://www.frontiersin.org/article/10.3389/fneur.2018.00349/full
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