In vivo gene‑editing research exploring CRISPR base editors to change HTT splicing; readers will find a preclinical study of a therapeutic strategy and delivery approach.
Key Takeaways
- Base editors were designed to disrupt the splice acceptor of HTT exon 13 to alter HTT splicing patterns
- AAV-delivered split-intein base editors in an HD rodent striatum reduced HTT fragment formation and aggregation
- Treatment improved functional deficits and attenuated brain atrophy in the treated Huntington's disease model