Webinar: Scalable Manufacturing and Nanovesicle Delivery of CRISPR-Cas9 Ribonucleoproteins Using a cGMP Compliant Cell Engineering Platform
Abstract:
CRISPR-Cas9 has tremendous potential as a therapeutic tool for treating human diseases. However, prolonged expression of the nuclease and gRNA from viral vectors in an in vivo context may cause unwanted off-target activity and immunogenicity. To overcome these safety issues, a system was recently developed for transient delivery of CRISPR-Cas9 ribonucleoprotein (RNP), recruiting Cas9 protein by chemically-induced dimerization and sgRNA via a viral RNA packaging signal into extracellular nanovesicles.
Broadcasted on September 17, 2020