Enhancing the efficiency of direct reprogramming of human primary fibroblasts into dopaminergic neuron-like cells through p53 suppression

Sci China Life Sci. 2014 Sep;57(9):867-75. doi: 10.1007/s11427-014-4730-2. Epub 2014 Aug 16.

Abstract

Dopaminergic (DA) neuron-like cells obtained through direct reprogramming of primary human fibroblasts offer exciting opportunities for treatment of Parkinson's disease. A significant obstacle is the low efficiency of conversion during the reprogramming process. Here, we demonstrate that the suppression of p53 significantly enhances the efficiency of transcription factor-mediated conversion of human fibroblasts into functional dopaminergic neurons. In particular, blocking p53 activity using a dominant-negative p53 (p53-DN) in IMR90 cells increases the conversion efficiency by 5-20 fold. The induced DA neuron-like cells exhibit dopamine neuron-specific gene expression, significant dopamine uptake and production capacities, and enables symptomatic relief in a rat Parkinson's disease model. Taken together, our findings suggest that p53 is a critical barrier in direct reprogramming of fibroblast into dopaminergic neurons.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Cellular Reprogramming*
  • Disease Models, Animal
  • Dopamine / metabolism*
  • Humans
  • Neurons / cytology*
  • Parkinson Disease / therapy
  • Rats
  • Rats, Sprague-Dawley
  • Real-Time Polymerase Chain Reaction
  • Tumor Suppressor Protein p53 / antagonists & inhibitors*

Substances

  • Tumor Suppressor Protein p53
  • Dopamine