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Concise Review: The Use of Stem Cells for Understanding and Treating Huntington's Disease

Bronwen Connor1

  • 1Department of Pharmacology and Clinical Pharmacology, Centre for Brain Research, School of Medical Science, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand.

Stem Cells (Dayton, Ohio)
|November 28, 2017
PubMed

Insights

Stem cell biology and cell reprogramming offer new hope for Huntington's disease (HD) research. These advances may lead to novel therapeutic strategies for this debilitating neurodegenerative disorder.

Area of Science:

  • Neuroscience
  • Genetics
  • Regenerative Medicine

Background:

  • Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG expansion mutation in the huntingtin (HTT) gene.
  • Despite the identification of the HTT gene two decades ago, therapeutic strategies for HD remain limited.

Purpose of the Study:

  • To review the role of compensatory neurogenesis in the HD brain.
  • To explore stem cell-based therapies for cell replacement and prevention of cell loss in HD.
  • To discuss advancements in cell reprogramming for modeling and treating HD.

Main Methods:

  • Review of recent literature on stem cell biology and cell reprogramming in the context of HD.
  • Discussion of compensatory neurogenesis in the HD brain.
  • Exploration of stem cell-based therapeutic approaches.
  • Analysis of cell reprogramming techniques for HD modeling and treatment.

Main Results:

  • Stem cell biology and cell reprogramming offer novel pathways to understand HD pathogenesis.
  • These technologies facilitate the identification and screening of potential drug targets.
  • Alternative donor cell sources for cell replacement therapy are being explored.
  • Genome editing advances complement these new technologies.

Conclusions:

  • Recent advances in stem cell biology and cell reprogramming present a promising new era for Huntington's disease research.
  • These technologies hold potential for developing effective therapeutic strategies to alleviate HD.
  • The integration of new technologies may lead to breakthroughs in treating this debilitating disorder.

Keywords:
Cell replacement therapyCompensatory neurogenesisDirect cell reprogrammingDisease modelingHuntington's diseaseInduced pluripotent stem cells

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