messenger RNA encoding Cas9

Messenger RNA encoding Cas9 is a key component of the CRISPR/Cas9-based gene insertion therapy being developed by Regeneron Pharmaceuticals and Intellia Therapeutics for the treatment of hemophilia B. This therapeutic approach utilizes a hybrid delivery system: a lipid nanoparticle (LNP) is used to deliver the mRNA encoding the Cas9 enzyme along with a specific guide RNA (gRNA) to the liver, while an adeno-associated virus (AAV) vector delivers a functional Factor IX (FIX) gene template. Once the LNP is internalized by hepatocytes, the mRNA is translated into the Cas9 protein, which then complexes with the gRNA to create a targeted double-strand break at the F9 locus in the patient's genome. The functional FIX template is subsequently integrated into the DNA via homology-directed repair (HDR), allowing for the permanent and stable endogenous production of Factor IX. This strategy is designed to provide a one-time, functional cure for patients with hemophilia B by restoring clotting factor levels to a therapeutic range.

Other names

Cas9 mRNACas-9 mRNACas 9 mRNAmRNA-encoded Cas9mRNA-Cas9mRNA-Cas-9mRNA-Cas 9