CRISPR-Cas9 knockin mice for genome editing and cancer modeling

Platt et al. (2014). Cell DOI: 10.1016/j.cell.2014.09.014 Citations: 1992

Parts used

SpCas9 NucleaseU6 Promoter

Canonical designs

U6-(gRNA)-hSyn-Cre-P2A-EGFP-KASH-hGHpA

Key findings

  • Generated first Cre-dependent Cas9 knockin mouse (Rosa26-LSL-Cas9). Enabled sgRNA-only AAV delivery for tissue-specific editing without dual vectors. Demonstrated multiplexed cancer modeling by simultaneously targeting Kras, p53, and Lkb1 with single AAV vector (AAV-KPL) containing three sgRNAs plus Cre recombinase.