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Items: 17

1.

PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1.

Wang GS, Kuyumcu-Martinez MN, Sarma S, Mathur N, Wehrens XH, Cooper TA.

J Clin Invest. 2009 Dec;119(12):3797-806. doi: 10.1172/JCI37976. Epub 2009 Nov 9.

2.

Pentamidine reverses the splicing defects associated with myotonic dystrophy.

Warf MB, Nakamori M, Matthys CM, Thornton CA, Berglund JA.

Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18551-6. doi: 10.1073/pnas.0903234106. Epub 2009 Oct 12.

3.

A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding.

Arambula JF, Ramisetty SR, Baranger AM, Zimmerman SC.

Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16068-73. doi: 10.1073/pnas.0901824106. Epub 2009 Sep 8.

4.

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.

Proc Natl Acad Sci U S A. 2009 Aug 18;106(33):13915-20. doi: 10.1073/pnas.0905780106. Epub 2009 Aug 10.

5.

Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA.

Wheeler TM, Sobczak K, Lueck JD, Osborne RJ, Lin X, Dirksen RT, Thornton CA.

Science. 2009 Jul 17;325(5938):336-9. doi: 10.1126/science.1173110.

6.

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.

7.

Rational and modular design of potent ligands targeting the RNA that causes myotonic dystrophy 2.

Lee MM, Pushechnikov A, Disney MD.

ACS Chem Biol. 2009 May 15;4(5):345-55. doi: 10.1021/cb900025w.

8.

RNA and disease.

Cooper TA, Wan L, Dreyfuss G.

Cell. 2009 Feb 20;136(4):777-93. doi: 10.1016/j.cell.2009.02.011. Review.

9.

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.

10.
11.

Myotonic dystrophy: RNA-mediated muscle disease.

Wheeler TM, Thornton CA.

Curr Opin Neurol. 2007 Oct;20(5):572-6. Review.

PMID:
17885447
12.

Trinucleotide repeat disorders.

Orr HT, Zoghbi HY.

Annu Rev Neurosci. 2007;30:575-621. Review.

PMID:
17417937
13.

Reversible model of RNA toxicity and cardiac conduction defects in myotonic dystrophy.

Mahadevan MS, Yadava RS, Yu Q, Balijepalli S, Frenzel-McCardell CD, Bourne TD, Phillips LH.

Nat Genet. 2006 Sep;38(9):1066-70. Epub 2006 Jul 30.

14.

Chemical modifiers of unstable expanded simple sequence repeats: what goes up, could come down.

Gomes-Pereira M, Monckton DG.

Mutat Res. 2006 Jun 25;598(1-2):15-34. Epub 2006 Feb 28. Review.

PMID:
16500684
15.

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.

16.

Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat.

Mankodi A, Logigian E, Callahan L, McClain C, White R, Henderson D, Krym M, Thornton CA.

Science. 2000 Sep 8;289(5485):1769-73.

17.

Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy.

Miller JW, Urbinati CR, Teng-Umnuay P, Stenberg MG, Byrne BJ, Thornton CA, Swanson MS.

EMBO J. 2000 Sep 1;19(17):4439-48.

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