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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Bibliografski detalji
Izdano u:J Pharmacol Exp Ther
Glavni autori: 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
Jezik:Inglês
Izdano: The American Society for Pharmacology and Experimental Therapeutics 2015
Teme:
Drug Discovery and Translational Medicine
Online pristup: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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