Therapeutic gene editing through delivery of CRISPR-Cas9-ribonucleoprotein (RNP) provides vast opportunities in treating genetic diseases. Several systemic administrations have been attempted through viral vector delivery in the past, but all have suffered from induced immunogenicity. CRISPR-Cas9 protein delivery has been recognized as an effectively permanent genomic DNA editing strategy due to its nonreplicable process offering improved specificity. However, one hurdle in the application of the CRISPR system for human medicine is <i>in vivo</i> delivery: there has been no systemic CRISPR-Cas-RNP delivery reported thus far. Herein we report the delivery of Cas9-RNP into mice through systemic tail-vein injection, achieving up to &gt;8% gene editing in liver and spleen.

<i>In vivo</i> gene editing in mice through systemic delivery of CRISPR/Cas9-ribonucleoprotein

256th National Meeting (2018)

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In vivo gene editing in mice through systemic delivery of CRISPR/Cas9-ribonucleoprotein