Prime-Editing

Programmable genome editors — CRISPR-Cas9, base editors and prime editors — delivered ex vivo or in vivo via lipid nanoparticles to treat genetic disease, from the first approved CRISPR therapy Casgevy to in-vivo redosable CRISPR and single-dose cardiovascular base editing.

Search-and-replace genome editing with a Cas9 nickase fused to a reverse transcriptase and an extended pegRNA — writing all 12 base substitutions and small indels without a double-strand break.