Daunorubicin reduces MBNL1 sequestration caused by CUG-repeat expansion and rescues cardiac dysfunctions in a Drosophila model of myotonic dystrophy

• Main Authors: Mouli Chakraborty (Author), Chantal Sellier (Author), Michel Ney (Author), Villa Pascal (Author), Nicolas Charlet-Berguerand (Author), Ruben Artero (Author), Beatriz Llamusi (Author)
• Formato: Livro
• Publicado em: The Company of Biologists, 2018-04-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Myotonic dystrophy (DM) is a dominantly inherited neuromuscular disorder caused by expression of mutant myotonin-protein kinase (DMPK) transcripts containing expanded CUG repeats. Pathogenic DMPK RNA sequesters the muscleblind-like (MBNL) proteins, causing alterations in metabolism of various RNAs....