Cas9-gM

Cas9-gM is an experimental CRISPR-Cas9 genome editing construct developed by AstraZeneca for the targeted disruption of the mouse *Pcsk9* (proprotein convertase subtilisin/kexin type 9) gene. It consists of the *Streptococcus pyogenes* Cas9 nuclease and a specific guide RNA, termed gM, which has perfect complementarity to exon 1 of the murine *Pcsk9* locus. Delivered via adenoviral vectors, the system induces double-strand breaks at the target site that are typically repaired by the error-prone non-homologous end-joining (NHEJ) pathway, resulting in frameshift mutations and gene knockout. In preclinical research using hypercholesterolemic mouse models, Cas9-gM is used to evaluate the efficacy and safety of gene-editing strategies and to study the role of PCSK9 in cholesterol homeostasis.

Other names

Cas9-gM adenoviral vectorCas-9-gM adenoviral vectorCas 9-gM adenoviral vectorCRISPR-Cas9 targeting mouse Pcsk9CRISPR-Cas-9 targeting mouse Pcsk9CRISPR-Cas 9 targeting mouse Pcsk9