Induced neural stem cells as a means of treatment in Huntington's disease

Expert Opin Biol Ther. 2017 Nov;17(11):1333-1343. doi: 10.1080/14712598.2017.1365133. Epub 2017 Aug 9.

Abstract

Huntington's disease (HD) is an inherited neurodegenerative disease characterized by chorea, dementia, and depression caused by progressive nerve cell degeneration, which is triggered by expanded CAG repeats in the huntingtin (Htt) gene. Currently, there is no cure for this disease, nor is there an effective medicine available to delay or improve the physical, mental, and behavioral severities caused by it. Areas covered: In this review, the authors describe the use of induced neural stem cells (iNSCs) by direct conversion technology, which offers great advantages as a therapeutic cell type to treat HD. Expert opinion: Cell conversion of somatic cells into a desired stem cell type is one of the most promising treatments for HD because it could be facilitated for the generation of patient-specific neural stem cells. The induced pluripotent stem cells (iPSCs) have a powerful potential for differentiation into neurons, but they may cause teratoma formation due to an undifferentiated pluripotent stem cell after transplantation Therefore, direct conversion of somatic cells into iNSCs is a promising alternative technology in regenerative medicine and the iNSCs may be provided as a therapeutic cell source for Huntington's disease.

Keywords: Huntington’s diseases; Induced neural stem cells; cell conversion; stem cell therapy.

Publication types

  • Review

MeSH terms

  • Cell Differentiation
  • Humans
  • Huntington Disease / pathology
  • Huntington Disease / therapy*
  • Induced Pluripotent Stem Cells / cytology
  • Induced Pluripotent Stem Cells / metabolism
  • Induced Pluripotent Stem Cells / transplantation*
  • Neural Stem Cells / cytology
  • Neural Stem Cells / metabolism
  • Neurons / cytology
  • Neurons / metabolism
  • Pluripotent Stem Cells / cytology
  • Pluripotent Stem Cells / transplantation
  • Teratoma / etiology
  • Transcription Factors / genetics
  • Transcription Factors / metabolism

Substances

  • Transcription Factors