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Abstract

Huntington’s disease is a hereditary neurodegenerative disease caused by the HTT gene mutation. While there are treatments for this disease, they are only known to temporarily alleviate symptoms, and have little effect on the progress of the disease. Clustered regularly interspaced short palindromic repeats (CRISPR), a genome editing technique, comprised of single guide RNSs (sgRNA) and single-stranded donor oligonucleotides (ssODN) repair template that can be used to edit and correct gene mutations in a variety of cells and organisms. In this study, CRISPR will be engineered to target and reduce the extended series of CAG repeats in the DNA that cause Huntington’s disease in C. elegans with the disease. The C. elegans will be injected with the sgRNA and ssODN repair template before day three and then allowed to reproduce. Following the injection and reproduction, their DNA and their offspring’s DNA will be sequenced to check the effectiveness of the CRISPR. This study is significant because it has the potential to correct the Huntington's gene mutation within an organism, therefore, ameliorating the symptoms.

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