Generation of induced pluripotent stem cell line(ZZUi006-A)from a patient with myotonic dystrophy type 1

Stem Cell Res. 2018 Oct:32:61-64. doi: 10.1016/j.scr.2018.08.013. Epub 2018 Aug 22.

Abstract

Myotonic dystrophy type 1 (DM1) is a dominantly inherited neurodegenerative disease caused by a CTG repeat expansion close to the DMPK gene. The fibroblasts from a patient with DM1 were successfully converted to induced pluripotent stem cells (iPSCs), designated ZZUi006-A, by employing episomal plasmids expressing OCT3/4, SOX2, KLF4, LIN28, L-MYC. The ZZUi006-A cell line may provide a robust platform for further study of DM1 pathogenesis as well as drug testing and gene therapy research.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Differentiation / genetics
  • Cell Differentiation / physiology
  • Cell Line
  • Cells, Cultured
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Induced Pluripotent Stem Cells / metabolism
  • Karyotype
  • Kruppel-Like Factor 4
  • Male
  • Microsatellite Repeats / genetics
  • Middle Aged
  • Myotonic Dystrophy / metabolism*
  • Polymerase Chain Reaction