Advancing Direct Enzyme Delivery for Therapeutic Genome Editing In Vivo

Genome-editing enzymes such as CRISPR-Cas9 have been successfully employed to target different genetic disorders in model systems, and ex vivo approaches have demonstrated the clinical potential of this technology. However, the challenge of delivering such corrective enzymes in vivo represents a substantial barrier to therapeutic translation. Viral vectors—refined during their use in traditional gene therapy—represent the most widely-used delivery platform in preclinical work, and nanoparticles are quickly maturing into a comparably powerful vehicle. Direct delivery of pre-formed CRISPR enzymes has recently emerged as an appealing strategy for enabling therapeutic editing in vivo. In this GEN webinar, we will hear how the Wilson lab works to advance this approach by performing molecular engineering, giving CRISPR enzymes the properties necessary for safe, efficient, and accessible delivery in a clinical setting. These efforts will enable in vivo delivery of CRISPR enzymes, including hard-to-deliver tools such as base editors and prime editors.