Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping.

P, Gee; MSY, Lung; Y, Okuzaki; N, Sasakawa; T, Iguchi; Y, Makita; H, Hozumi; Y, Miura; LF, Yang; M, Iwasaki; XH, Wang; MA, Waller; N, Shirai; YO, Abe; Y, Fujita; K, Watanabe; A, Kagita; KA, Iwabuchi; M, Yasuda; H, Xu; T, Noda; J, Komano; H, Sakurai; N, Inukai; A, Hotta

doi:10.1038/s41467-020-14957-y

Public

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping.

Shared by NobleBlocks on Mar 13, 2020 • 12:00 AM UTC

Authors:

Gee P

Lung MSY

Okuzaki Y

Abstract

Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is needed for therapeutic genome editing applications. Here, we develop an extracellular nanovesicle-based ribonucleoprotein delivery sy...

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