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Article

RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington’s disease models

13 Jan 2025

Abstract excerpt

BACKGROUND: HD is a devastating neurodegenerative disorder caused by the expansion of CAG repeats in the HTT. Silencing the expression of mutated proteins is a therapeutic direction to rescue HD patients, and recent advances in gene editing technology such as CRISPR/CasRx have opened up new avenues for therapeutic intervention. METHODS: The CRISPR/CasRx system was employed to target human HTT exon 1, resulting in...

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PMID: 39806441
DOI: 10.1186/s13024-024-00794-w

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