The Delivery Challenge of Genome Editing in Human Epithelia

Despite exciting advances in gene editing, their clinical translation is still hampered by the lack of delivery systems that can encapsulate and deliver gene editing tools like CRISPR-Cas9 or prime editors to the target side. This is particularly challenging in human epithelia, such as the skin and the lung; the latter of which being a mucosal surface that is covered by a mucus layer. In this perspective, the design and biological assessment of delivery systems for gene editing tools like CRISPR in skin and mucosal surfaces are discussed. The current state-of-the-art, current knowledge, and translational gaps, and guide toward improved translation are highlighted.

Automated summary

This article discusses the design and biological assessment of delivery systems for gene editing tools like CRISPR in skin and mucosal surfaces, highlighting the current state-of-the-art, current knowledge, and translational gaps towards improved translation.