Manumycin A corrects aberrant splicing of Clcn1 in myotonic dystrophy type 1 (DM1) mice
Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy in adults and as yet no cure for DM1. Here, we report the potential of manumycin A for a novel DM1 therapeutic reagent. DM1 is caused by expansion of CTG repeat. Mutant transcripts containing expanded CUG repeats lead to aberrant...
Main Authors: | , , , , , , |
---|---|
Format: | Online |
Language: | English |
Published: |
Nature Publishing Group
2013
|
Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3701899/ |