Myoblasts generated by lentiviral mediated MyoD transduction of myotonic dystrophy type 1 (DM1) fibroblasts can be used for assays of therapeutic molecules

BMC Research Notes

Volumn 4, Issue , 2011, Pages

  Larsen, Jan ^a   Pettersson, Olof J ^a   Jakobsen, Maria ^a   Thomsen, Rune ^a   Pedersen, Christina B ^b   Hertz, Jens M ^c   Gregersen, Niels ^b   Corydon, Thomas J ^a   Jensen, Thomas G ^a  

^a AARHUS UNIVERSITY   (Denmark)

^b AARHUS UNIVERSITY HOSPITAL   (Denmark)

^c ODENSE UNIVERSITY HOSPITAL   (Denmark)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 81055148308     PISSN: None     EISSN: 17560500     Source Type: Journal    
DOI: 10.1186/1756-0500-4-490     Document Type: Article

References (25)

1. Pathogenic mechanisms of myotonic dystrophy. Lee JE, Cooper TA, Biochem Soc Trans 2009 37 1281 1286
2. Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3' end of a transcript encoding a protein kinase family member. Brook JD, McCurrach ME, Harley HG, Buckler AJ, Church D, Aburatani H, Hunter K, Stanton VP, Thirion JP, Hudson T, Sohn R, Zemelman B, Snell RG, Rundle SA, Crow S, Davies J, Shelbourne P, Buxton J, Jones C, Juvonen V, Johnson K, Harper PS, Shaw DJ, Housman DE, Cell 1992 68 799 808
3. Aberrant alternative splicing and extracellular matrix gene expression in mouse models of myotonic dystrophy. Du H, Cline MS, Osborne RJ, Tuttle DL, Clark TA, Donohue JP, Hall MP, Shiue L, Swanson MS, Thornton CA, Ares M Jr, Nat Struct Mol Biol 2010 17 187 193
4. Disruption of splicing regulated by a CUG-binding protein in myotonic dystrophy. Philips AV, Timchenko LT, Cooper TA, Science 1998 280 737 741 (Pubitemid 28243348)
5. Transgenic mice expressing CUG-BP1 reproduce splicing mis-regulation observed in myotonic dystrophy. Ho TH, Bundman D, Armstrong DL, Cooper TA, Hum Mol Genet 2005 14 1539 1547 (Pubitemid 40823462)
6. Pentamidine reverses the splicing defects associated with myotonic dystrophy. Warf MB, Nakamori M, Matthys CM, Thornton CA, Berglund JA, Proc Natl Acad Sci USA 2009 106 18551 18556
7. 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 USA 2009 106 13915 13920
8. Expansion of a CUG trinucleotide repeat in the 3' untranslated region of myotonic dystrophy protein kinase transcripts results in nuclear retention of transcripts. Davis BM, McCurrach ME, Taneja KL, Singer RH, Housman DE, Proc Natl Acad Sci USA 1997 94 7388 7393
9. Expression of a single transfected cDNA converts fibroblasts to myoblasts. Davis RL, Weintraub H, Lassar AB, Cell 1987 51 987 1000
10. 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 4 9 18
11. Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy cells. Chaouch S, Mouly V, Goyenvalle A, Vulin A, Mamchaoui K, Negroni E, Di Santo J, Butler-Browne G, Torrente Y, Garcia L, Furling D, Hum Gene Ther 2009 20 784 790
12. 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 325 336 339
13. Amelioration of psoriasis by anti-TNF-alpha RNAi in the xenograft transplantation model. Jakobsen M, Stenderup K, Rosada C, Moldt B, Kamp S, Dam TN, Jensen TG, Mikkelsen JG, Mol Ther 2009 17 1743 1753
14. SingerLab Protocols. http://www.singerlab.org/protocols/insitu-mammalian. htm
15. Localization of trinucleotide repeat sequences in myotonic dystrophy cells using a single fluorochrome-labeled PNA probe. Taneja KL, Biotechniques 1998 24 472 476 (Pubitemid 28115129)
16. DNA/polyethyleneimine/hyaluronic acid small complex particles and tumor suppression in mice. Ito T, Yoshihara C, Hamada K, Koyama Y, Biomaterials 2010 31 2912 2918
17. Promoter-specific regulation of MyoD binding and signal transduction cooperate to pattern gene expression. Bergstrom DA, Penn BH, Strand A, Perry RL, Rudnicki MA, Tapscott SJ, Mol Cell 2002 9 587 600 (Pubitemid 34273789)
18. MBNL3/CHCR prevents myogenic differentiation by inhibiting MyoD-dependent gene transcription. Lee KS, Smith K, Amieux PS, Wang EH, Differentiation 2008 76 299 309 (Pubitemid 351322682)
19. Mutant DMPK 3'-UTR transcripts disrupt C2C12 myogenic differentiation by compromising MyoD. Amack JD, Reagan SR, Mahadevan MS, J Cell Biol 2002 159 419 429 (Pubitemid 35332829)
20. Normal myogenesis and increased apoptosis in myotonic dystrophy type-1 muscle cells. Loro E, Rinaldi F, Malena A, Masiero E, Novelli G, Angelini C, Romeo V, Sandri M, Botta A, Vergani L, Cell Death Differ 2010 17 1315 1324
21. Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2. Mankodi A, Urbinati CR, Yuan QP, Moxley RT, Sansone V, Krym M, Henderson D, Schalling M, Swanson MS, Thornton CA, Hum Mol Genet 2001 10 2165 2170 (Pubitemid 32998828)
22. Increased steady-state levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyperphosphorylation. Kuyumcu-Martinez NM, Wang GS, Cooper TA, Mol Cell 2007 28 68 78 (Pubitemid 47531966)
23. Myotonic dystrophy type 1 is associated with nuclear foci of mutant RNA, sequestration of muscleblind proteins and deregulated alternative splicing in neurons. Jiang H, Mankodi A, Swanson MS, Moxley RT, Thornton CA, Hum Mol Genet 2004 13 3079 3088 (Pubitemid 40045262)
24. In vivo co-localisation of MBNL protein with DMPK expanded-repeat transcripts. Fardaei M, Larkin K, Brook JD, Hamshere MG, Nucleic Acids Res 2001 29 2766 2771 (Pubitemid 32685039)
25. Aberrant regulation of insulin receptor alternative splicing is associated with insulin resistance in myotonic dystrophy. Savkur RS, Philips AV, Cooper TA, Nat Genet 2001 29 40 47 (Pubitemid 32801806)