Therapeutic in vivo delivery of gene editing agents

In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements. Therapeutic gene editing has made exciting progress in the past few years, in part through key advances in in vivo delivery technologies. David Liu and colleagues review the essential characteristics required for efficient in vivo gene editing delivery vehicles, discussing the pros and cons of viral vectors, lipid nanoparticles, and virus-like particles.