ApoB-targeting gRNA VLP

ApoB-targeting gRNA VLP refers to a gene-editing therapeutic approach that utilizes engineered virus-like particles (eVLPs) to deliver CRISPR/Cas9 ribonucleoproteins (RNPs) specifically to the liver. The system consists of a Cas9 protein complexed with a guide RNA (gRNA) designed to target and disrupt the *APOB* gene. By inducing permanent genetic insertions or deletions (indels) in the *APOB* locus, the therapy aims to reduce the production of Apolipoprotein B, a critical structural component of atherogenic lipoproteins like LDL and VLDL. This approach is being developed primarily for the treatment of atherosclerotic cardiovascular disease (ASCVD) and familial hypercholesterolemia. The technology, notably advanced by researchers at the Broad Institute and Harvard University (e.g., David Liu's lab) and commercialized by Nvelop Therapeutics, offers a potentially safer alternative to viral vectors by providing transient expression of the gene-editing machinery, thereby minimizing off-target effects and immunogenicity.

Other names

eVLP-Cas9-sgApobeVLP-Cas-9-sgApobeVLP-Cas 9-sgApobApoB-targeting eVLPengineered virus-like particles targeting Apob