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Items: 1 to 20 of 124

1.

Design of a bioactive small molecule that targets the myotonic dystrophy type 1 RNA via an RNA motif-ligand database and chemical similarity searching.

Parkesh R, Childs-Disney JL, Nakamori M, Kumar A, Wang E, Wang T, Hoskins J, Tran T, Housman D, Thornton CA, Disney MD.

J Am Chem Soc. 2012 Mar 14;134(10):4731-42. doi: 10.1021/ja210088v.

2.

Rationally designed small molecules targeting the RNA that causes myotonic dystrophy type 1 are potently bioactive.

Childs-Disney JL, Hoskins J, Rzuczek SG, Thornton CA, Disney MD.

ACS Chem Biol. 2012 May 18;7(5):856-62. doi: 10.1021/cb200408a.

3.

Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3.

Pushechnikov A, Lee MM, Childs-Disney JL, Sobczak K, French JM, Thornton CA, Disney MD.

J Am Chem Soc. 2009 Jul 22;131(28):9767-79. doi: 10.1021/ja9020149.

4.

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.

5.

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.

6.

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.

7.

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.

PMID:
16723374
8.

Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules.

Childs-Disney JL, Stepniak-Konieczna E, Tran T, Yildirim I, Park H, Chen CZ, Hoskins J, Southall N, Marugan JJ, Patnaik S, Zheng W, Austin CP, Schatz GC, Sobczak K, Thornton CA, Disney MD.

Nat Commun. 2013;4:2044. doi: 10.1038/ncomms3044.

9.

Molecular Effects of the CTG Repeats in Mutant Dystrophia Myotonica Protein Kinase Gene.

Llamusí B, Artero R.

Curr Genomics. 2008 Dec;9(8):509-16. doi: 10.2174/138920208786847944.

10.

Chemical correction of pre-mRNA splicing defects associated with sequestration of muscleblind-like 1 protein by expanded r(CAG)-containing transcripts.

Kumar A, Parkesh R, Sznajder LJ, Childs-Disney JL, Sobczak K, Disney MD.

ACS Chem Biol. 2012 Mar 16;7(3):496-505. doi: 10.1021/cb200413a.

11.

HTS-Compatible Patient-Derived Cell-Based Assay to Identify Small Molecule Modulators of Aberrant Splicing in Myotonic Dystrophy Type 1.

O'Leary DA, Vargas L, Sharif O, Garcia ME, Sigal YJ, Chow SK, Schmedt C, Caldwell JS, Brinker A, Engels IH.

Curr Chem Genomics. 2010 Mar 19;4:9-18. doi: 10.2174/1875397301004010009.

12.

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.

13.

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.

14.

Controlling the specificity of modularly assembled small molecules for RNA via ligand module spacing: targeting the RNAs that cause myotonic muscular dystrophy.

Lee MM, Childs-Disney JL, Pushechnikov A, French JM, Sobczak K, Thornton CA, Disney MD.

J Am Chem Soc. 2009 Dec 2;131(47):17464-72. doi: 10.1021/ja906877y.

15.

Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1.

Jahromi AH, Fu Y, Miller KA, Nguyen L, Luu LM, Baranger AM, Zimmerman SC.

J Med Chem. 2013 Dec 12;56(23):9471-81. doi: 10.1021/jm400794z.

16.

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.

17.

Molecular mechanisms responsible for aberrant splicing of SERCA1 in myotonic dystrophy type 1.

Hino S, Kondo S, Sekiya H, Saito A, Kanemoto S, Murakami T, Chihara K, Aoki Y, Nakamori M, Takahashi MP, Imaizumi K.

Hum Mol Genet. 2007 Dec 1;16(23):2834-43.

PMID:
17728322
18.

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.

19.

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.

20.

Rationally designed small molecules that target both the DNA and RNA causing myotonic dystrophy type 1.

Nguyen L, Luu LM, Peng S, Serrano JF, Chan HY, Zimmerman SC.

J Am Chem Soc. 2015 Nov 11;137(44):14180-9. doi: 10.1021/jacs.5b09266.

PMID:
26473464
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