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Gene Therapy · us

Instead of Shutting Down HTT, Rewrite Its Splicing Path: Base Editing Alleviates Huntington’s Disease Pathology in Mice

The research team altered the splice acceptor of HTT exon 13, enabling the protein to bypass a cleavage site prone to generating toxic fragments. Brain atrophy and multiple functional deficits were subsequently reduced in mice, but challenges involving brain delivery and long-term safety still stand between the therapy and human trials.