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 par-ticles. 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.

Automated summary

In vivo gene editing therapies have the potential to treat the underlying causes of genetic diseases. This review examines current delivery technologies for therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. The benefits and drawbacks of each method are compared, and opportunities for future improvements are highlighted.