PLLF + Cas9-sgDAD1

PLLF + Cas9-sgDAD1 is an experimental gene therapy nanoparticle system designed for the treatment of bladder cancer, particularly non-muscle-invasive bladder cancer. It consists of fluorinated polylysine (PLLF) micelles that serve as a delivery vehicle for a CRISPR-Cas9 plasmid targeting the defender against cell death 1 (DAD1) gene. DAD1 is an anti-apoptotic gene frequently overexpressed in bladder cancer. By delivering the Cas9-sgDAD1 system transmucosally via intravesical administration, the therapy induces a knockout of DAD1, which triggers cancer cell apoptosis through the MAPK signaling pathway. Preclinical studies in orthotopic bladder cancer mouse models have demonstrated significant therapeutic efficacy and a lack of systemic toxicity.