activated protein C AAV knock-in vector

The **activated protein C AAV knock-in vector** is an investigational in vivo gene editing therapy developed by researchers at Jichi Medical University, Kyoto Prefectural University of Medicine, and the University of Tokyo. It is designed for the treatment of severe congenital protein C deficiency, a life-threatening neonatal thrombotic disorder that causes purpura fulminans. The therapy utilizes a single adeno-associated virus (AAV) vector to deliver a compact CRISPR-Cas effector, engineered AsCas12f (enAsCas12f), along with a guide RNA targeting intron 13 of the albumin (*Alb*) locus and a donor template encoding an engineered activated protein C (APC). By utilizing non-homologous end joining (NHEJ)-mediated knock-in, the vector enables liver-specific integration and sustained expression of functional anticoagulant APC, preventing lethal thrombosis and rescuing lethality in neonatal models of severe protein C deficiency.

Other names

AAV-enAsCas12f-activated protein C knock-in vectorAAV-enAsCas-12f-activated protein C knock-in vectorAAV-enAsCas 12f-activated protein C knock-in vectorsingle AAV vector-mediated enAsCas12f knock-in genome editing of activated protein C