Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic diseases including myotonic dystrophy (DM1). Thus, ASO strategies for DM1 can abolish the toxic RNA gain-of-function mechanism caused by nuclear-retained mutant transcripts conta...

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Bibliographic Details
Main Authors:	Klein, AF, Varela-Muino, MA, Arandel, L, Holland, A, Naouar, N, Arzumanov, A, Seoane, D, Revillod, L, Bassez, G, Ferry, A, Jauvin, D, Gourdon, G, Puymirat, J, Gait, MJ, Furling, D, Wood, MJA
Format:	Journal article
Language:	English
Published:	American Society for Clinical Investigation 2019

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