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Cells of Matter—In Vitro Models for Myotonic Dystrophy

Archive ouverte | Matloka, Magdalena | CCSD

International audience. Myotonic dystrophy type 1 (DM1 also known as Steinert disease) is a multisystemic disorder mainly characterized by myotonia, progressive muscle weakness and wasting, cognitive impairments, an...

New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeats

Archive ouverte | Laurent, François-Xavier | CCSD

International audience. Myotonic Dystrophy type I (DM1) is caused by an abnormal expansion of CTG triplets in the 3 0 UTR of the dystrophia myotonica protein kinase (DMPK) gene, leading to the aggregation of the mut...

Low-dose of peptide-conjugate antisense oligonucleotides targeting CUGexp-RNA in murine skeletal muscles normalizes Myotonic Dystrophy 1 phenotype

Archive ouverte | Klein, Arnaud F. | CCSD

International audience. Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic diseases including Myotonic Dystrophy type 1 (DM1). DM1 is a dominant...

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