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Adv Healthc Mater · 2021

The Delivery Challenge of Genome Editing in Human Epithelia.

Advanced healthcare materials, 10(19): e2100847 PMID 34165913 DOI 10.1002/adhm.202100847

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Abstract

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.

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