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Abstract
CRISPR-Cas9 technology offers transformative potential in treating Huntington's Disease by addressing its genetic root causes.
- Huntington's Disease is characterized by toxic mutant huntingtin protein resulting from expanded CAG repeats in the HTT gene.
- Current therapeutic approaches face limitations in effectively targeting the genetic basis of the disease.
- CRISPR-based strategies may effectively excise expanded repeats and target specific alleles in preclinical studies using animal models and human cells.
- Delivery methods, including viral and non-viral vectors, are critical in optimizing therapeutic outcomes and minimizing unintended effects.
- Ethical and safety considerations, particularly regarding precision and long-term impacts of gene editing, are essential for advancing these therapies.
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