Cooperative transcription activation by Nurr1 and Pitx3 induces embryonic stem cell maturation to the midbrain dopamine neuron phenotype

Proc Natl Acad Sci U S A. 2006 Feb 21;103(8):2874-9. doi: 10.1073/pnas.0511153103. Epub 2006 Feb 13.

Abstract

Midbrain dopamine (DA) neurons play a central role in the regulation of voluntary movement, and their degeneration is associated with Parkinson's disease. Cell replacement therapies, and in particular embryonic stem (ES) cell-derived DA neurons, offer a potential therapeutic venue for Parkinson's disease. We sought to identify genes that can potentiate maturation of ES cell cultures to the midbrain DA neuron phenotype. A number of transcription factors have been implicated in the development of midbrain DA neurons by expression analyses and loss-of-function knockout mouse studies, including Nurr1, Pitx3, Lmx1b, Engrailed-1, and Engrailed-2. However, none of these factors appear sufficient alone to induce the mature midbrain DA neuron phenotype in ES cell cultures in vitro, suggesting a more complex regulatory network. Here we show that Nurr1 and Pitx3 cooperatively promote terminal maturation to the midbrain DA neuron phenotype in murine and human ES cell cultures.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Differentiation / genetics*
  • Cells, Cultured
  • DNA-Binding Proteins / metabolism
  • DNA-Binding Proteins / physiology*
  • Dopamine / metabolism
  • Embryo, Mammalian / cytology
  • Gene Expression Regulation, Developmental*
  • Homeodomain Proteins / metabolism
  • Homeodomain Proteins / physiology*
  • Humans
  • Mesencephalon / cytology
  • Mesencephalon / growth & development*
  • Mesencephalon / metabolism
  • Mice
  • Neurons / cytology
  • Neurons / metabolism
  • Nuclear Receptor Subfamily 4, Group A, Member 2
  • Phenotype
  • Stem Cell Transplantation
  • Stem Cells / cytology*
  • Transcription Factors / metabolism
  • Transcription Factors / physiology*

Substances

  • DNA-Binding Proteins
  • Homeodomain Proteins
  • NR4A2 protein, human
  • Nr4a2 protein, mouse
  • Nuclear Receptor Subfamily 4, Group A, Member 2
  • Transcription Factors
  • homeobox protein PITX3
  • Dopamine