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...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:J Pharmacol Exp Ther
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: The American Society for Pharmacology and Experimental Therapeutics 2015
Θέματα:
Drug Discovery and Translational Medicine
Διαθέσιμο Online: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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