Cell-based therapies for Huntington's disease

Drug Discov Today. 2014 Jul;19(7):980-4. doi: 10.1016/j.drudis.2014.02.012. Epub 2014 Mar 12.

Abstract

Cell-based therapies are a viable option for the long-term treatment of Huntington's disease (HD), which is characterized by progressive neurodegeneration predominately in the striatum and cortex. Current research focuses on genetic suppression of the mutant huntingtin (mHTT) gene and cell replacement therapy of the lost cells in HD. As we discuss here, the recent development of induced pluripotent stem (iPS) cells technology demonstrated the potential of cell-based therapy in rodent models. It was shown that iPSCs were capable of differentiating into lost neurons in HD and stem cell grafts can improve motor deficiency in HD rodent models. Altogether, these findings have shown great promise for developing the foundation of the cell-based therapy.

Publication types

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

MeSH terms

  • Animals
  • Cell- and Tissue-Based Therapy / methods*
  • Genetic Therapy / methods*
  • Humans
  • Huntingtin Protein
  • Huntington Disease / diagnosis
  • Huntington Disease / genetics*
  • Huntington Disease / therapy*
  • Induced Pluripotent Stem Cells / transplantation
  • Nerve Tissue Proteins / antagonists & inhibitors
  • Nerve Tissue Proteins / genetics

Substances

  • HTT protein, human
  • Huntingtin Protein
  • Nerve Tissue Proteins