Toward an age of CRISPR delivery with non-viral biologics

A major goal in CRISPR/Cas gene therapy delivery is achieving biocompatible, transient delivery of gene editing machinery directly in patients. Among emerging delivery options, extracellular vesicles (EVs) hold promise as a new class of non-viral biologically derived vehicles for biotherapeutic applications for in vivo applications.1 Synthetic nanoparticles, such as lipid nanoparticles (LNPs), have empowered the non-viral delivery of CRISPR/Cas systems including a recent unprecedented advance in gene editing with the correction of a patient-specific genetic mutation in a rare disease (the “N = 1” patient).