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Using gene or cell therapies to treat Huntington's disease
Caroline S Binda1, Mariah J Lelos2, Anne E Rosser3
1Division of Psychological Medicine and Clinical Neurosciences, School of Medicine, Cardiff University, Cardiff, United Kingdom; UK Dementia Research Institute at Cardiff, Cardiff University, Cardiff, United Kingdom.
Handbook of Clinical Neurology
|September 28, 2024
Summary
Huntington's disease (HD) stems from a genetic mutation causing toxic protein buildup. Current research explores gene and cell therapies to slow progression and repair neural damage in affected individuals.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG repeat expansion in the HTT gene.
- This expansion leads to toxic mutant huntingtin protein, transcriptional dysregulation, and disrupted cellular metabolism.
- The striatum is primarily affected early in HD, preceding neurological symptoms.
Purpose of the Study:
- To review the current status of therapeutic strategies for Huntington's disease.
- To discuss the prospects and challenges of gene and cell therapies.
- To explore potential combination therapies for disease modification and neural repair.
Main Methods:
- Review of existing literature on Huntington's disease pathogenesis and therapeutic approaches.
- Analysis of gene-targeting strategies (DNA/RNA level) and cell replacement therapies.
- Consideration of genetic modifiers influencing disease onset and progression.
Main Results:
- No definitive disease-modifying treatments are currently available for HD.
- Therapeutics targeting DNA and RNA pathways are advancing, with some in human trials.
- Cell therapies offer potential for neural cell replacement and striatal support.
Conclusions:
- Gene and cell therapies represent promising avenues for treating Huntington's disease.
- Combining gene and cell therapies may offer a dual approach to slow disease and promote neural repair.
- Further research is needed to overcome challenges and optimize these therapeutic strategies.
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