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Items: 1 to 20 of 254


RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1.

Lee JE, Bennett CF, Cooper TA.

Proc Natl Acad Sci U S A. 2012 Mar 13;109(11):4221-6. doi: 10.1073/pnas.1117019109. Epub 2012 Feb 27.


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.

Nature. 2012 Aug 2;488(7409):111-5. doi: 10.1038/nature11362.


Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy.

Leger AJ, Mosquea LM, Clayton NP, Wu IH, Weeden T, Nelson CA, Phillips L, Roberts E, Piepenhagen PA, Cheng SH, Wentworth BM.

Nucleic Acid Ther. 2013 Apr;23(2):109-17. doi: 10.1089/nat.2012.0404. Epub 2013 Jan 11.


RNA interference targeting CUG repeats in a mouse model of myotonic dystrophy.

Sobczak K, Wheeler TM, Wang W, Thornton CA.

Mol Ther. 2013 Feb;21(2):380-7. doi: 10.1038/mt.2012.222. Epub 2012 Nov 27.


Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy.

Wojtkowiak-Szlachcic A, Taylor K, Stepniak-Konieczna E, Sznajder LJ, Mykowska A, Sroka J, Thornton CA, Sobczak K.

Nucleic Acids Res. 2015 Mar 31;43(6):3318-31. doi: 10.1093/nar/gkv163. Epub 2015 Mar 9.


Expanded CTG repeats within the DMPK 3' UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy.

Orengo JP, Chambon P, Metzger D, Mosier DR, Snipes GJ, Cooper TA.

Proc Natl Acad Sci U S A. 2008 Feb 19;105(7):2646-51. doi: 10.1073/pnas.0708519105. Epub 2008 Feb 13.


Stabilization of expanded (CTG)•(CAG) repeats by antisense oligonucleotides.

Nakamori M, Gourdon G, Thornton CA.

Mol Ther. 2011 Dec;19(12):2222-7. doi: 10.1038/mt.2011.191. Epub 2011 Oct 4.


Ribonuclear foci at the neuromuscular junction in myotonic dystrophy type 1.

Wheeler TM, Krym MC, Thornton CA.

Neuromuscul Disord. 2007 Mar;17(3):242-7. Epub 2007 Feb 15.


Cytoplasmic CUG RNA foci are insufficient to elicit key DM1 features.

Dansithong W, Wolf CM, Sarkar P, Paul S, Chiang A, Holt I, Morris GE, Branco D, Sherwood MC, Comai L, Berul CI, Reddy S.

PLoS One. 2008;3(12):e3968. doi: 10.1371/journal.pone.0003968. Epub 2008 Dec 18.


MBNL1 and CUGBP1 modify expanded CUG-induced toxicity in a Drosophila model of myotonic dystrophy type 1.

de Haro M, Al-Ramahi I, De Gouyon B, Ukani L, Rosa A, Faustino NA, Ashizawa T, Cooper TA, Botas J.

Hum Mol Genet. 2006 Jul 1;15(13):2138-45. Epub 2006 May 24.


Elevation of RNA-binding protein CUGBP1 is an early event in an inducible heart-specific mouse model of myotonic dystrophy.

Wang GS, Kearney DL, De Biasi M, Taffet G, Cooper TA.

J Clin Invest. 2007 Oct;117(10):2802-11.


In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models.

García-López A, Llamusí B, Orzáez M, Pérez-Payá E, Artero RD.

Proc Natl Acad Sci U S A. 2011 Jul 19;108(29):11866-71. doi: 10.1073/pnas.1018213108. Epub 2011 Jul 5.


DDX6 regulates sequestered nuclear CUG-expanded DMPK-mRNA in dystrophia myotonica type 1.

Pettersson OJ, Aagaard L, Andrejeva D, Thomsen R, Jensen TG, Damgaard CK.

Nucleic Acids Res. 2014 Jun;42(11):7186-200. doi: 10.1093/nar/gku352. Epub 2014 May 3.


Age of onset of RNA toxicity influences phenotypic severity: evidence from an inducible mouse model of myotonic dystrophy (DM1).

Gladman JT, Mandal M, Srinivasan V, Mahadevan MS.

PLoS One. 2013 Sep 5;8(9):e72907. doi: 10.1371/journal.pone.0072907. eCollection 2013.


Myotonic dystrophy associated expanded CUG repeat muscleblind positive ribonuclear foci are not toxic to Drosophila.

Houseley JM, Wang Z, Brock GJ, Soloway J, Artero R, Perez-Alonso M, O'Dell KM, Monckton DG.

Hum Mol Genet. 2005 Mar 15;14(6):873-83. Epub 2005 Feb 9.


Muscleblind-like 1 knockout mice reveal novel splicing defects in the myotonic dystrophy brain.

Suenaga K, Lee KY, Nakamori M, Tatsumi Y, Takahashi MP, Fujimura H, Jinnai K, Yoshikawa H, Du H, Ares M Jr, Swanson MS, Kimura T.

PLoS One. 2012;7(3):e33218. doi: 10.1371/journal.pone.0033218. Epub 2012 Mar 13.


Sense and Antisense DMPK RNA Foci Accumulate in DM1 Tissues during Development.

Michel L, Huguet-Lachon A, Gourdon G.

PLoS One. 2015 Sep 4;10(9):e0137620. doi: 10.1371/journal.pone.0137620. eCollection 2015.


HnRNP H inhibits nuclear export of mRNA containing expanded CUG repeats and a distal branch point sequence.

Kim DH, Langlois MA, Lee KB, Riggs AD, Puymirat J, Rossi JJ.

Nucleic Acids Res. 2005 Jul 15;33(12):3866-74. Print 2005.


Colocalization of muscleblind with RNA foci is separable from mis-regulation of alternative splicing in myotonic dystrophy.

Ho TH, Savkur RS, Poulos MG, Mancini MA, Swanson MS, Cooper TA.

J Cell Sci. 2005 Jul 1;118(Pt 13):2923-33. Epub 2005 Jun 16.


Gain of RNA function in pathological cases: Focus on myotonic dystrophy.

Klein AF, Gasnier E, Furling D.

Biochimie. 2011 Nov;93(11):2006-12. doi: 10.1016/j.biochi.2011.06.028. Epub 2011 Jul 13. Review.


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