Description
Priority frontier extracted from landscape commentary: in vivo prime editing for therapeutic applications.
Resolution criteria
Resolution requires: (1) In vivo prime editing with >=20% correction of target allele in disease model; (2) Off-target validation showing <=2% off-targets at top-50 sites; (3) Prime editor variant with >=2-fold improved efficiency; (4) Preclinical safety profile at therapeutic dose. Deliverable: therapeutic proof-of-concept + safety profile.
Evidence summary
in vivo prime editing for therapeutic applications