Description
Priority frontier extracted from landscape commentary: CRISPR delivery systems for in vivo therapeutic applications.
Resolution criteria
Resolution requires: (1) CRISPR delivery system achieving >=40% editing efficiency in >=2 target tissues in vivo; (2) Off-target analysis showing <=1% indel frequency at top-10 off-target sites; (3) Delivery validated for cargo up to 10kb; (4) Standardized characterization protocol adopted by >=1 consortium. Deliverable: delivery system + characterization protocol.
Evidence summary
CRISPR delivery systems for in vivo therapeutic applications