Identification and Characterization of Modified Antisense Oligonucleotides Targeting DMPK in Mice and Nonhuman Primates for the Treatment of Myotonic Dystrophy Type 1

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy in adults. DM1 is caused by an expanded CTG repeat in the 3′-untranslated region of DMPK, the gene encoding dystrophia myotonica protein kinase (DMPK). Antisense oligonucleotides (ASOs) containing 2′,4′-constrained ethyl-m...

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Detalhes bibliográficos
Publicado no:J Pharmacol Exp Ther
Main Authors: Pandey, Sanjay K., Wheeler, Thurman M., Justice, Samantha L., Kim, Aneeza, Younis, Husam S., Gattis, Danielle, Jauvin, Dominic, Puymirat, Jack, Swayze, Eric E., Freier, Susan M., Bennett, C. Frank, Thornton, Charles A., MacLeod, A. Robert
Formato: Artigo
Idioma:Inglês
Publicado em: The American Society for Pharmacology and Experimental Therapeutics 2015
Assuntos:
Drug Discovery and Translational Medicine
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4613955/
https://ncbi.nlm.nih.gov/pubmed/26330536
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1124/jpet.115.226969
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