Generation of patient-specific induced neuronal cells using a direct reprogramming strategy

Stem Cells Dev. 2014 Jan 1;23(1):16-23. doi: 10.1089/scd.2013.0131. Epub 2013 Sep 17.


Direct reprogramming of human fibroblasts into functional neurons in vitro by defined factors provides an invaluable resource for regenerative medicine. However, clinical applications must consider the risk of immune rejection, thus patient-specific induced neuronal cells (iNCs) may serve as an ideal source for autologous cell replacement. In this study, we report a robust process for functional neuronal cells from the patients' scalp by lentiviral gene delivery of Ascl1, Myt1l, and Sox2. These three-factor iNCs are similar to human neuronal cells in morphology, surface antigens, gene expression, and electrophysiological characteristics. Our findings might provide a source of patient-specific functional neurons for cell therapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Basic Helix-Loop-Helix Transcription Factors / genetics
  • Brain Injuries / therapy
  • Cell Differentiation
  • Cell Transformation, Neoplastic
  • Cell- and Tissue-Based Therapy*
  • Cells, Cultured
  • Cellular Reprogramming / genetics*
  • Fibroblasts / physiology*
  • Gene Expression
  • Gene Expression Regulation, Developmental
  • Homeodomain Proteins / genetics
  • Humans
  • Ion Channel Gating
  • Ion Channels / analysis
  • Mice
  • Mice, SCID
  • Nerve Regeneration
  • Nerve Tissue Proteins / genetics
  • Neural Stem Cells / physiology*
  • Neurons / physiology*
  • POU Domain Factors / genetics
  • SOXB1 Transcription Factors / genetics
  • Skin / cytology
  • Transcription Factors / genetics


  • ASCL1 protein, human
  • Basic Helix-Loop-Helix Transcription Factors
  • Homeodomain Proteins
  • Ion Channels
  • MYT1L protein, human
  • Nerve Tissue Proteins
  • POU Domain Factors
  • SOX2 protein, human
  • SOXB1 Transcription Factors
  • Transcription Factors
  • transcription factor Brn-2