890
Views
13
CrossRef citations to date
0
Altmetric
Addendum

GSK3β is a new therapeutic target for myotonic dystrophy type 1

, , &
Article: e26555 | Received 31 Jul 2013, Accepted 20 Sep 2013, Published online: 26 Sep 2013

References

  • Harper PS. Myotonic Dystrophy. London: WB Saunders, 2001.
  • Brook JD, McCurrach ME, Harley HG, Buckler AJ, Church D, Aburatani H, Hunter K, Stanton VP, Thirion JP, Hudson T, et al. Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3′ end of a transcript encoding a protein kinase family member. Cell 1992; 68:799 - 808; http://dx.doi.org/10.1016/0092-8674(92)90154-5; PMID: 1310900
  • Fu YH, Pizzuti A, Fenwick RG Jr., King J, Rajnarayan S, Dunne PW, Dubel J, Nasser GA, Ashizawa T, de Jong P, et al. An unstable triplet repeat in a gene related to myotonic muscular dystrophy. Science 1992; 255:1256 - 8; http://dx.doi.org/10.1126/science.1546326; PMID: 1546326
  • Timchenko LT. Myotonic dystrophy: the role of RNA CUG triplet repeats. Am J Hum Genet 1999; 64:360 - 4; http://dx.doi.org/10.1086/302268; PMID: 9973273
  • Mankodi A, Logigian E, Callahan L, McClain C, White R, Henderson D, Krym M, Thornton CA. Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat. Science 2000; 289:1769 - 73; http://dx.doi.org/10.1126/science.289.5485.1769; PMID: 10976074
  • Seznec H, Agbulut O, Sergeant N, Savouret C, Ghestem A, Tabti N, Willer JC, Ourth L, Duros C, Brisson E, et al. Mice transgenic for the human myotonic dystrophy region with expanded CTG repeats display muscular and brain abnormalities. Hum Mol Genet 2001; 10:2717 - 26; http://dx.doi.org/10.1093/hmg/10.23.2717; PMID: 11726559
  • Ranum LP, Cooper TA. RNA-mediated neuromuscular disorders. Annu Rev Neurosci 2006; 29:259 - 77; http://dx.doi.org/10.1146/annurev.neuro.29.051605.113014; PMID: 16776586
  • Schoser B, Timchenko L. Myotonic dystrophies 1 and 2: complex diseases with complex mechanisms. Curr Genomics 2010; 11:77 - 90; http://dx.doi.org/10.2174/138920210790886844; PMID: 20885816
  • Timchenko LT, Timchenko NA, Caskey CT, Roberts R. Novel proteins with binding specificity for DNA CTG repeats and RNA CUG repeats: implications for myotonic dystrophy. Hum Mol Genet 1996; 5:115 - 21; http://dx.doi.org/10.1093/hmg/5.1.115; PMID: 8789448
  • Timchenko LT, Miller JW, Timchenko NA, DeVore DR, Datar KV, Lin L, Roberts R, Caskey CT, Swanson MS. Identification of a (CUG)n triplet repeat RNA-binding protein and its expression in myotonic dystrophy. Nucleic Acids Res 1996; 24:4407 - 14; http://dx.doi.org/10.1093/nar/24.22.4407; PMID: 8948631
  • Miller JW, Urbinati CR, Teng-Umnuay P, Stenberg MG, Byrne BJ, Thornton CA, Swanson MS. Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy. EMBO J 2000; 19:4439 - 48; http://dx.doi.org/10.1093/emboj/19.17.4439; PMID: 10970838
  • Taneja KL, McCurrach M, Schalling M, Housman D, Singer RH. Foci of trinucleotide repeat transcripts in nuclei of myotonic dystrophy cells and tissues. J Cell Biol 1995; 128:995 - 1002; http://dx.doi.org/10.1083/jcb.128.6.995; PMID: 7896884
  • Kanadia RN, Johnstone KA, Mankodi A, Lungu C, Thornton CA, Esson D, Timmers AM, Hauswirth WW, Swanson MS. A muscleblind knockout model for myotonic dystrophy. Science 2003; 302:1978 - 80; http://dx.doi.org/10.1126/science.1088583; PMID: 14671308
  • Timchenko NA, Cai Z-J, Welm AL, Reddy S, Ashizawa T, Timchenko LT. RNA CUG repeats sequester CUGBP1 and alter protein levels and activity of CUGBP1. J Biol Chem 2001; 276:7820 - 6; http://dx.doi.org/10.1074/jbc.M005960200; PMID: 11124939
  • Savkur RS, Philips AV, Cooper TA. Aberrant regulation of insulin receptor alternative splicing is associated with insulin resistance in myotonic dystrophy. Nat Genet 2001; 29:40 - 7; http://dx.doi.org/10.1038/ng704; PMID: 11528389
  • Kuyumcu-Martinez NM, Wang GS, Cooper TA. Increased steady-state levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyperphosphorylation. Mol Cell 2007; 28:68 - 78; http://dx.doi.org/10.1016/j.molcel.2007.07.027; PMID: 17936705
  • Huichalaf C, Sakai K, Jin B, Jones K, Wang G-L, Schoser B, Schneider-Gold C, Sarkar P, Pereira-Smith OM, Timchenko N, et al. Expansion of CUG RNA repeats causes stress and inhibition of translation in myotonic dystrophy 1 (DM1) cells. FASEB J 2010; 24:3706 - 19; http://dx.doi.org/10.1096/fj.09-151159; PMID: 20479119
  • Timchenko NA, Wang G-L, Timchenko LT. RNA CUG-binding protein 1 increases translation of 20-kDa isoform of CCAAT/enhancer-binding protein beta by interacting with the alpha and beta subunits of eukaryotic initiation translation factor 2. J Biol Chem 2005; 280:20549 - 57; http://dx.doi.org/10.1074/jbc.M409563200; PMID: 15788409
  • Timchenko LT, Salisbury E, Wang G-L, Nguyen H, Albrecht JH, Hershey JWB, Timchenko NA. Age-specific CUGBP1-eIF2 complex increases translation of CCAAT/enhancer-binding protein beta in old liver. J Biol Chem 2006; 281:32806 - 19; http://dx.doi.org/10.1074/jbc.M605701200; PMID: 16931514
  • Anderson P, Kedersha N. RNA granules. J Cell Biol 2006; 172:803 - 8; http://dx.doi.org/10.1083/jcb.200512082; PMID: 16520386
  • Paillard L, Omilli F, Legagneux V, Bassez T, Maniey D, Osborne HB. EDEN and EDEN-BP, a cis element and an associated factor that mediate sequence-specific mRNA deadenylation in Xenopus embryos. EMBO J 1998; 17:278 - 87; http://dx.doi.org/10.1093/emboj/17.1.278; PMID: 9427761
  • Philips AV, Timchenko LT, Cooper TA. Disruption of splicing regulated by a CUG-binding protein in myotonic dystrophy. Science 1998; 280:737 - 41; http://dx.doi.org/10.1126/science.280.5364.737; PMID: 9563950
  • Charlet-B N, Savkur RS, Singh G, Philips AV, Grice EA, Cooper TA, Charlet-BN. Loss of the muscle-specific chloride channel in type 1 myotonic dystrophy due to misregulated alternative splicing. Mol Cell 2002; 10:45 - 53; http://dx.doi.org/10.1016/S1097-2765(02)00572-5; PMID: 12150906
  • Rattenbacher B, Beisang D, Wiesner DL, Jeschke JC, von Hohenberg M, St Louis-Vlasova IA, Bohjanen PR. Analysis of CUGBP1 targets identifies GU-repeat sequences that mediate rapid mRNA decay. Mol Cell Biol 2010; 30:3970 - 80; http://dx.doi.org/10.1128/MCB.00624-10; PMID: 20547756
  • Jones K, Wei C, Iakova P, Bugiardini E, Schneider-Gold C, Meola G, Woodgett J, Killian J, Timchenko NA, Timchenko LT. GSK3β mediates muscle pathology in myotonic dystrophy. J Clin Invest 2012; 122:4461 - 72; http://dx.doi.org/10.1172/JCI64081; PMID: 23160194
  • Naderi S, Gutzkow KB, Låhne HU, Lefdal S, Ryves WJ, Harwood AJ, Blomhoff HK. cAMP-induced degradation of cyclin D3 through association with GSK-3beta. J Cell Sci 2004; 117:3769 - 83; http://dx.doi.org/10.1242/jcs.01210; PMID: 15252116
  • Wheeler TM, Sobczak K, Lueck JD, Osborne RJ, Lin X, Dirksen RT, Thornton CA. Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA. Science 2009; 325:336 - 9; http://dx.doi.org/10.1126/science.1173110; PMID: 19608921
  • 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, et al. Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy. Proc Natl Acad Sci U S A 2009; 106:13915 - 20; http://dx.doi.org/10.1073/pnas.0905780106; PMID: 19667189
  • Wheeler TM, Leger AJ, Pandey SK, MacLeod AR, Nakamori M, Cheng SH, Wentworth BM, Bennett CF, Thornton CA. Targeting nuclear RNA for in vivo correction of myotonic dystrophy. Nature 2012; 488:111 - 5; http://dx.doi.org/10.1038/nature11362; PMID: 22859208
  • Doble BW, Woodgett JR. GSK-3: tricks of the trade for a multi-tasking kinase. J Cell Sci 2003; 116:1175 - 86; http://dx.doi.org/10.1242/jcs.00384; PMID: 12615961