Stem cell-based cell therapy for Huntington disease: a review
Manho Kim1, Soon-Tae Lee, Kon Chu
1Department of Neurology, Seoul National University College of Medicine, Seoul, Korea.
Summary
Huntington disease (HD) lacks effective treatments. Neural stem cell therapy shows promise in animal models, offering potential for future Huntington disease therapies.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Stem Cell Biology
Background:
- Huntington disease (HD) is a severe neurodegenerative disorder with no current cure.
- Fetal neural transplantation has shown limited efficacy in treating HD.
- Advances in stem cell biology offer new therapeutic avenues for HD.
Purpose of the Study:
- To review the current status of stem cell therapy in experimental Huntington disease models.
- To discuss the potential of neural stem cells for future HD therapeutic strategies.
Main Methods:
- Review of preclinical and clinical studies on cell therapy for Huntington disease.
- Focus on the use of embryonic stem cells, bone marrow mesenchymal stem cells, and neural stem cells.
- Analysis of neural stem cell differentiation and functional recovery in HD models.
Main Results:
- Neural stem cells are preferred for experimental HD studies due to their differentiation capacity.
- Intracerebral or intravenous transplantation of neural stem cells has induced functional recovery in HD models.
- Various stem cell types have been explored in animal models of neurological disorders.
Conclusions:
- Neural stem cells demonstrate significant potential for treating Huntington disease.
- Further research into neural stem cell-based therapies is warranted for developing effective Huntington disease treatments.
Related Concept Videos
iPS Cell Differentiation
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
Huntington Disease l: Introduction
Huntington disease or HD is a progressive, fatal neurodegenerative disorder inherited in an autosomal dominant pattern.PathophysiologyIt is caused by expansion of the CAG trinucleotide repeat in the HTT gene on chromosome 4 (4p16.3), producing an abnormal huntingtin protein with an expanded polyglutamine tract. This misfolded protein disrupts cellular function, leading to neuronal death. Normal alleles have ≤26 repeats, 27–35 are intermediate (risk of expansion), 36–39 show reduced penetrance,...
EPS and iPS Cells in Disease Research
Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
Stem Cell Culture
Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
Stem Cell Therapy for Tissue Regeneration
Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
Induced Pluripotent Stem Cells
Stem cells are undifferentiated cells that divide and produce different types of cells. Ordinarily, cells that have differentiated into a specific cell type are post-mitotic—that is, they no longer divide. However, scientists have found a way to reprogram these mature cells so that they “de-differentiate” and return to an unspecialized, proliferative state. These cells are also pluripotent like embryonic stem cells—able to produce all cell types—and are therefore called induced pluripotent stem...


