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Targeting nuclear RNA for in vivo correction of myotonic dystrophy.
Wheeler TM, Leger AJ, Pandey SK, MacLeod AR, Nakamori M, Cheng SH, Wentworth BM, Bennett CF, Thornton CA. Wheeler TM, et al. Nature. 2012 Aug 2;488(7409):111-5. doi: 10.1038/nature11362. Nature. 2012. PMID: 22859208 Free PMC article.
Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy.
Mulders SA, van den Broek WJ, Wheeler TM, Croes HJ, van Kuik-Romeijn P, de Kimpe SJ, Furling D, Platenburg GJ, Gourdon G, Thornton CA, Wieringa B, Wansink DG. Mulders SA, et al. Proc Natl Acad Sci U S A. 2009 Aug 18;106(33):13915-20. doi: 10.1073/pnas.0905780106. Epub 2009 Aug 10. Proc Natl Acad Sci U S A. 2009. PMID: 19667189 Free PMC article.
Splicing biomarkers of disease severity in myotonic dystrophy.
Nakamori M, Sobczak K, Puwanant A, Welle S, Eichinger K, Pandya S, Dekdebrun J, Heatwole CR, McDermott MP, Chen T, Cline M, Tawil R, Osborne RJ, Wheeler TM, Swanson MS, Moxley RT 3rd, Thornton CA. Nakamori M, et al. Ann Neurol. 2013 Dec;74(6):862-72. doi: 10.1002/ana.23992. Ann Neurol. 2013. PMID: 23929620 Free PMC article.
Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1.
Pandey SK, Wheeler TM, Justice SL, Kim A, Younis HS, Gattis D, Jauvin D, Puymirat J, Swayze EE, Freier SM, Bennett CF, Thornton CA, MacLeod AR. Pandey SK, et al. J Pharmacol Exp Ther. 2015 Nov;355(2):329-40. doi: 10.1124/jpet.115.226969. Epub 2015 Sep 1. J Pharmacol Exp Ther. 2015. PMID: 26330536 Free PMC article.