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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 nucleus-retained mutant DMPK (DM1 protein...

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Detalles Bibliográficos
Autores principales:	Klein, Arnaud F., Varela, Miguel A., Arandel, Ludovic, Holland, Ashling, Naouar, Naira, Arzumanov, Andrey, Seoane, David, Revillod, Lucile, Bassez, Guillaume, Ferry, Arnaud, Jauvin, Dominic, Gourdon, Genevieve, Puymirat, Jack, Gait, Michael J., Furling, Denis, Wood, Matthew J.A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2019
Materias:	Concise Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6819114/ https://www.ncbi.nlm.nih.gov/pubmed/31479430 http://dx.doi.org/10.1172/JCI128205

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