Year: 2025
Type: article
Abstract: Huntington’s disease is genetic neurodegenerative disorder that usually appears in individuals between the ages of 30 and 50, causing profound impairments in motor function, cognition, and emotional regulation. The underlying etiology of this condition is linked to mutations in the DNA that lead to the abnormal aggregation of the huntingtin protein, ultimately causing neuronal cell death. In the absence of effective treatment options, pertinent research continues to investigate gene editing technology as a potential therapeutic approach. This paper focuses on the application of CRISPR-Cas9 technology, which aims to precisely edit the mutated huntingtin gene, particularly targeting and disrupting the CAG repeat expansion to mitigate neurodegeneration and restore normal gene function. Through the literature review and case analyses, it evaluates the efficacy of CRISPR-Cas9 in mitigating huntingtin protein aggregation and enhancing patient prognosis. Therefore, the results suggest that CRISPR technology holds promise as an innovative therapeutic approach for Huntington’s disease. Nevertheless, challenges such as off-target effects and efficient gene delivery need to be addressed. In short, therapeutic strategies like CRISPR, antisense oligonucleotides (ASOs), and small interfering RNAs (siRNAs) show considerable promise for driving future research advancements.
Source: Theoretical and Natural Science
Author: Junyi Fan
Institution: Sichuan Agricultural University
Language: English
Subfield: Cellular and Molecular Neuroscience
Field: Neuroscience
Domain: Life Sciences
Open Access status: hybrid
OpenAlex ID: W4407823535