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Updated: Aug 26, 2025

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
Published on: May 2, 2025
Pathogenesis and potential therapeutic application of stem cells transplantation in Huntington's disease
Sudeepto Saha1, Manami Jayati Dey2, Salman Khan Promon1
1Department of Life Sciences, School of Environment and Life Sciences, Independent University, Bangladesh (IUB), Dhaka, Bangladesh.
Insights
Huntington's disease (HD) is a fatal neurodegenerative disorder. Stem cell therapy shows promise as a potential cure by addressing the underlying cause, offering hope beyond symptom management.
Area of Science:
- Neuroscience
- Genetics
- Regenerative Medicine
Background:
- Huntington's disease (HD) is a progressive, fatal neurodegenerative disorder.
- Caused by CAG/glutamine repeats in the Huntington gene, leading to movement, cognitive, and affective impairments.
- Current treatments only manage symptoms, not the disease's root cause.
Purpose of the Study:
- To review the pathogenesis of Huntington's disease.
- To evaluate the efficacy and clinical practicality of stem cell transplantation for HD.
- To analyze the application of stem cell therapy in genetically altered HD animal models.
Main Methods:
- Literature review focusing on Huntington's disease pathogenesis.
- Analysis of studies on stem cell transplantation in HD.
- Examination of data from genetically modified animal models of HD.
Main Results:
- Stem cell therapy presents a potential breakthrough for treating Huntington's disease.
- Research in animal models is crucial for understanding HD pathogenesis and therapeutic potential.
- The review analyzes the application and effectiveness of stem cell transplantation in preclinical HD models.
Conclusions:
- Stem cell transplantation offers a promising therapeutic avenue for Huntington's disease.
- Further research and clinical application in animal models are essential.
- This approach could lead to a cure by targeting the disease's underlying genetic cause.
Abstract:
Huntington's disease (HD) is a progressive neurodegenerative disorder which is caused due to repetitive CAG or glutamine expression along the coding region of the Huntington gene. This disease results in certain movement abnormalities, affective disturbances, dementia and cognitive impairments. To this date, there is no proper cure for this rare and fatal neurological condition but there have been certain advancements in the field of genetic animal model research studies to elucidate the understanding of the pathogenesis of this condition. Currently, HD follows a certain therapeutic approach which just relieves the symptoms but doesn't cure the underlying cause of the disease. Stem cell therapy can be a breakthrough in developing a potential cure for this condition. In this review, we have discussed the pathogenesis and the efficacy and clinical practicality of the therapeutic application of stem cell transplantation in Huntington's disease. The application of this groundbreaking therapy on genetically altered animal models has been listed and analyzed in brief.
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