Comparison of Non-viral Delivery Vehicles for CRISPR/Cas9 Therapies

Abstract

CRISPR/Cas9 is a gene editing tool that is rapidly replacing previous technologies, such as ZFNs and TALENS, to rectify disease-causing mutations. Oftentimes, these diseases have treatment methods that target the symptoms rather than the cause. Recent innovations in bioengineering suggest that novel gene therapies may provide a better alternative than existing treatments, by correcting the cause of the disorder directly: the genomic DNA. The excitement around gene therapy, however, is abated by the challenges in how to deliver the technology. Currently, there are two methods of delivery, viral and non-viral vectors, of which non-viral vectors are considered safer and more practical. Such non-viral carriers include gold nanoparticles, lipid nanoparticles, and polymeric carriers, of which will be the focus of this paper. This review examines the efficacy of these existing non-viral carriers through a comprehensive literature analysis. We compare the percentage of cells showing the targeted change in vivo and in vitro across the different vehicles in an attempt to understand how efficacy changes across vectors. Overall, we highlight that the optimal delivery platform is likely dependent on the disease model and target tissue. In the future, researchers can use this analysis to assess currently available designs and develop new carriers for transporting CRISPR/Cas9 to specific targets in vivo and in vitro.