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ABSTRACT
Introduction: 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.
Article highlights
Huntington’s disease (HD) is a neurodegenerative disorder, and HD patients present loss of GABAergic medium spiny neurons (MSNs) in striatum.
Since no therapeutic agents have been developed to treat HD, the cell-based therapies that replace lost neurons can be a new therapeutic approach.
Neural stem cells (NSCs) are capable of self-renewing and differentiating into three neural cell types: neurons, astrocytes and oligodendrocytes. Therefore, iNSCs may be provided to replace the damaged GABAergic MSNs of HD patients.
Human somatic cells, such as MSC, astrocytes and fibroblasts, can be directly converted into iNSCs, which may facilitate the development of personalized stem-cell-based therapy for HD.
Induced NSCs that have been converted without use of foreign DNA can be used as therapeutic cells for HD treatment.
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Declaration of interest
The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.