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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Veröffentlicht in:J Pharmacol Exp Ther
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: The American Society for Pharmacology and Experimental Therapeutics 2015
Schlagworte:
Drug Discovery and Translational Medicine
Online Zugang: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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