DNA and RNA editing for the therapy of human diseases: current status, challenges, and future prospects.

Mol Biomed · 2026 · PMC13103161 · PMID 42018092

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In chronic infection, AAV-delivered SaCas9 targeting the HBV genome achieved detectable in vivo editing in humanized liver mouse models of chronic HBV infection and, on a background of nucleos(t)ide analogue therapy, showed a trend toward reduced intrahepatic HBV DNA/cccDNA with good tolerability—providing in vivo support for curative strategies aimed at cccDNA [ 94 ].

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