When CASGEVY, the first CRISPR-based therapy, was approved by the FDA in December, it gave CRISPR technology the validation it needed. While the research around using CRISPR for neurodegenerative diseases is still very much in the early stages, Huntington’s disease (HD) has been identified as a perfect candidate for CRISPR gene editing, because it's an inherited neurodegenerative disorder caused by a single mutation and the presence of an abnormal protein.
One of the most promising CRISPR studies for HD comes from researchers at Jinan University in Guangzhou, China, who were able to demonstrate that CRISPR-Cas9 editing can be used to correct the mutation in HTT, replacing the hyperexpansion with a normal CAG repeat. Preclinical research has been promising and could hopefully progress into clinical trials. Click here to learn more.
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