CRISPR-Cas9 Pcsk9 deletion

CRISPR-Cas9 Pcsk9 deletion is a gene-editing therapeutic approach that utilizes the CRISPR-Cas9 system to introduce permanent loss-of-function mutations or deletions in the *PCSK9* gene. Primarily targeted at hepatocytes, this disruption reduces the production of the PCSK9 protein, which typically facilitates the degradation of low-density lipoprotein receptors (LDLR). By increasing LDLR density on the cell surface, the therapy enhances the clearance of LDL-cholesterol from the plasma, offering a potential treatment for hypercholesterolemia and familial hypercholesterolemia. Additionally, preclinical research has identified PCSK9 as a target for cancer immunotherapy; its deletion prevents the lysosomal degradation of MHC class I molecules, thereby boosting tumor cell surface MHC I expression and enhancing CD8+ T-cell-mediated anti-tumor immunity. This platform is being explored by several entities, including CRISPR Therapeutics (CTX310, CTX330) and AccurEdit Therapeutics (ART002).

Other names

PCSK9 gene editingPCSK-9 gene editingPCSK 9 gene editingCRISPR-mediated PCSK9 disruptionPcsk9 depletionPcsk-9 depletionPcsk 9 depletion