title: Myoblasts generated by lentiviral mediated MyoD transduction of myotonic dystrophy type 1 (DM1) fibroblasts can be used for assays of therapeutic molecules.
abstract: BACKGROUND
  Myotonic dystrophy type 1 (DM1) is the most common muscle dystrophy in adults. The disease is caused by a triplet expansion in the 3'end of the myotonic dystrophy protein kinase (DMPK) gene. In order to develop a human cell model for investigation of possible effects of antisense and RNAi effector molecules we have used lentiviral mediated myoD-forced myogenesis of DM1 patient fibroblasts.
  
  
  RESULTS
  Transduced fibroblasts show a multinuclear phenotype and express the differentiation marker myogenin. Furthermore, fluorescence in situ hybridization (FISH) analysis revealed a statistical significant increase in the amount of nuclear foci in DM1 patient fibroblasts after myogenesis. Finally, no nuclear foci were found after treatment with oligonucleotides targeting the repeat expansions.
  
  
  CONCLUSIONS
  The abundance of nuclear foci in DM1 patient fibroblasts increase following myogenesis, as visualized by FISH analysis. Foci were eradicated after treatment with antisense oligonucleotides. Thus, we propose that the current cell model is suitable for testing of novel treatment modalities.
authors.affiliation: Department of Biomedicine, Aarhus University, Aarhus, Denmark. thomas@humgen.au.dk.
authors.forename: Jan
authors.initials: J
authors.lastname: Larsen
authors.forename: Olof J
authors.initials: OJ
authors.lastname: Pettersson
authors.forename: Maria
authors.initials: M
authors.lastname: Jakobsen
authors.forename: Rune
authors.initials: R
authors.lastname: Thomsen
authors.forename: Christina B
authors.initials: CB
authors.lastname: Pedersen
authors.forename: Jens M
authors.initials: JM
authors.lastname: Hertz
authors.forename: Niels
authors.initials: N
authors.lastname: Gregersen
authors.forename: Thomas J
authors.initials: TJ
authors.lastname: Corydon
authors.forename: Thomas G
authors.initials: TG
authors.lastname: Jensen
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