Stem cell therapy and cellular engineering for treatment of neuronal dysfunction in Huntington's disease

Kyung Ah Choi, Insik Hwang, Hang soo Park, Seung Ick Oh, Seongman Kang, Sunghoi Hong

Research output: Contribution to journalReview articlepeer-review

8 Citations (Scopus)


Huntington's disease (HD) is a fatal inherited neurodegenerative disorder characterized by progressive loss of neurons in the striatum, a sub-cortical region of the forebrain. The sub-cortical region of the forebrain is associated with the control of movement and behavior, thus HD initially presents with coordination difficulty and cognitive decline. Recent reprogramming technologies, including induced pluripotent stem cells (iPSCs) and induced neural stem cells (iNSCs), have created opportunities to understand the pathological cascades that underlie HD and to develop new treatments for this currently incurable neurological disease. The ultimate objectives of stem cell-based therapies for HD are to replace lost neurons and to prevent neuronal dysfunction and death. In this review, we examine the current understanding of the molecular and pathological mechanisms involved in HD. We discuss disease modeling with HD-iPSCs derived from the somatic cells of patients, which could provide an invaluable platform for understanding HD pathogenesis. We speculate about the benefits and drawbacks of using iNSCs as an alternative stem cell source for HD treatment. Finally, we discuss cell culture and engineering systems that promote the directed differentiation of pluripotent stem cell-derived NSCs into a striatal DARPP32+ GABAergic MSN phenotype for HD. In conclusion, this review summarizes the potentials of cell reprogramming and engineering technologies relevant to the development of cell-based therapies for HD.

Original languageEnglish
Pages (from-to)882-894
Number of pages13
JournalBiotechnology Journal
Issue number7
Publication statusPublished - 2014 Jul


  • Cell therapy
  • Human cell model
  • Huntington's disease
  • Medium spiny neuron
  • Reprogramming technology

ASJC Scopus subject areas

  • Applied Microbiology and Biotechnology
  • Molecular Medicine


Dive into the research topics of 'Stem cell therapy and cellular engineering for treatment of neuronal dysfunction in Huntington's disease'. Together they form a unique fingerprint.

Cite this