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From dynamic combinatorial 'hit' to lead: in vitro and in vivo activity of compounds targeting the pathogenic RNAs that cause myotonic dystrophy.

Ofori LO, Hoskins J, Nakamori M, Thornton CA, Miller BL.

Nucleic Acids Res. 2012 Jul;40(13):6380-90. doi: 10.1093/nar/gks298. Epub 2012 Apr 6.


Dynamic combinatorial selection of molecules capable of inhibiting the (CUG) repeat RNA-MBNL1 interaction in vitro: discovery of lead compounds targeting myotonic dystrophy (DM1).

Gareiss PC, Sobczak K, McNaughton BR, Palde PB, Thornton CA, Miller BL.

J Am Chem Soc. 2008 Dec 3;130(48):16254-61. doi: 10.1021/ja804398y.


Targeting toxic RNAs that cause myotonic dystrophy type 1 (DM1) with a bisamidinium inhibitor.

Wong CH, Nguyen L, Peh J, Luu LM, Sanchez JS, Richardson SL, Tuccinardi T, Tsoi H, Chan WY, Chan HY, Baranger AM, Hergenrother PJ, Zimmerman SC.

J Am Chem Soc. 2014 Apr 30;136(17):6355-61. doi: 10.1021/ja5012146. Epub 2014 Apr 22.


Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy.

Kanadia RN, Shin J, Yuan Y, Beattie SG, Wheeler TM, Thornton CA, Swanson MS.

Proc Natl Acad Sci U S A. 2006 Aug 1;103(31):11748-53. Epub 2006 Jul 24.


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.


RNA Foci, CUGBP1, and ZNF9 are the primary targets of the mutant CUG and CCUG repeats expanded in myotonic dystrophies type 1 and type 2.

Jones K, Jin B, Iakova P, Huichalaf C, Sarkar P, Schneider-Gold C, Schoser B, Meola G, Shyu AB, Timchenko N, Timchenko L.

Am J Pathol. 2011 Nov;179(5):2475-89. doi: 10.1016/j.ajpath.2011.07.013. Epub 2011 Sep 1.


Modifications to toxic CUG RNAs induce structural stability, rescue mis-splicing in a myotonic dystrophy cell model and reduce toxicity in a myotonic dystrophy zebrafish model.

deLorimier E, Coonrod LA, Copperman J, Taber A, Reister EE, Sharma K, Todd PK, Guenza MG, Berglund JA.

Nucleic Acids Res. 2014 Nov 10;42(20):12768-78. doi: 10.1093/nar/gku941. Epub 2014 Oct 10.


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.


Expanded CUG repeats Dysregulate RNA splicing by altering the stoichiometry of the muscleblind 1 complex.

Paul S, Dansithong W, Jog SP, Holt I, Mittal S, Brook JD, Morris GE, Comai L, Reddy S.

J Biol Chem. 2011 Nov 4;286(44):38427-38. doi: 10.1074/jbc.M111.255224. Epub 2011 Sep 7.


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 is the primary determinant of focus formation and aberrant insulin receptor splicing in DM1.

Dansithong W, Paul S, Comai L, Reddy S.

J Biol Chem. 2005 Feb 18;280(7):5773-80. Epub 2004 Nov 16. Erratum in: J Biol Chem. 2005 May 20;280(20):20176.


Muscleblind protein, MBNL1/EXP, binds specifically to CHHG repeats.

Kino Y, Mori D, Oma Y, Takeshita Y, Sasagawa N, Ishiura S.

Hum Mol Genet. 2004 Mar 1;13(5):495-507. Epub 2004 Jan 13.


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.


CAG repeats mimic CUG repeats in the misregulation of alternative splicing.

Mykowska A, Sobczak K, Wojciechowska M, Kozlowski P, Krzyzosiak WJ.

Nucleic Acids Res. 2011 Nov 1;39(20):8938-51. doi: 10.1093/nar/gkr608. Epub 2011 Jul 27.


New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeats.

Laurent FX, Sureau A, Klein AF, Trouslard F, Gasnier E, Furling D, Marie J.

Nucleic Acids Res. 2012 Apr;40(7):3159-71. doi: 10.1093/nar/gkr1228. Epub 2011 Dec 9.


Overexpression of MBNL1 fetal isoforms and modified splicing of Tau in the DM1 brain: two individual consequences of CUG trinucleotide repeats.

Dhaenens CM, Schraen-Maschke S, Tran H, Vingtdeux V, Ghanem D, Leroy O, Delplanque J, Vanbrussel E, Delacourte A, Vermersch P, Maurage CA, Gruffat H, Sergeant A, Mahadevan MS, Ishiura S, Buée L, Cooper TA, Caillet-Boudin ML, Charlet-Berguerand N, Sablonnière B, Sergeant N.

Exp Neurol. 2008 Apr;210(2):467-78. doi: 10.1016/j.expneurol.2007.11.020. Epub 2007 Dec 4.


Transcriptional and post-transcriptional impact of toxic RNA in myotonic dystrophy.

Osborne RJ, Lin X, Welle S, Sobczak K, O'Rourke JR, Swanson MS, Thornton CA.

Hum Mol Genet. 2009 Apr 15;18(8):1471-81. doi: 10.1093/hmg/ddp058. Epub 2009 Feb 17.


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.


The role of flexibility in the rational design of modularly assembled ligands targeting the RNAs that cause the myotonic dystrophies.

Disney MD, Lee MM, Pushechnikov A, Childs-Disney JL.

Chembiochem. 2010 Feb 15;11(3):375-82. doi: 10.1002/cbic.200900716.


Novel proteins with binding specificity for DNA CTG repeats and RNA CUG repeats: implications for myotonic dystrophy.

Timchenko LT, Timchenko NA, Caskey CT, Roberts R.

Hum Mol Genet. 1996 Jan;5(1):115-21.


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