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유전체 교정 연구단
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Rationally designed nanoparticle delivery of Cas9 ribonucleoprotein for effective gene editing

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Title
Rationally designed nanoparticle delivery of Cas9 ribonucleoprotein for effective gene editing
Author(s)
Chae, Se-Youl; Euihwan Jeong; Kang, Seounghun; Yim, Yeajee; Jin-Soo Kim; Min, Dal-Hee
Publication Date
2022-05
Journal
Journal of Controlled Release, v.345, pp.108 - 119
Publisher
Elsevier B.V.
Abstract
© 2022 Elsevier B.V.Programmable endonucleases such as CRISPR/Cas9 system emerge as a promising tool to treat genetic and non-genetic diseases such as hypercholesterolemia, Duchenne muscular dystrophy, and cancer. However, the lack of safe and efficient vehicles that enable intracellular delivery of CRISPR/Cas9 endonuclease is a big hurdle for its therapeutic applications. Here, we employed porous nanoparticle for the Cas9 ribonucleoprotein (RNP) delivery and achieved efficient knockout of target genes in vitro and in vivo. The porous nanoparticle, called ‘BALL’, enabled safe and direct intracellular Cas9 RNP delivery by improving bioavailability and serum stability. The BALL-mediated delivery of Cas9 RNP showed superior indel efficiency of about 40% in vitro and 20% in vivo in a model system employing green fluorescent protein (GFP). More importantly, intramuscular injection of the Cas9 RNP-BALL complex targeting the myostatin (MSTN) gene which is known to suppress muscle growth achieved successful knockout of the MSTN gene, resulting in the increase of muscle and the improved motor functions. Thus, we believe that the BALL is a promising delivery system for CRISPR-based genome editing technology, which can be applied to the treatment of various genetic diseases.
URI
https://pr.ibs.re.kr/handle/8788114/11503
DOI
10.1016/j.jconrel.2022.02.035
ISSN
0168-3659
Appears in Collections:
Center for Genome Engineering(유전체 교정 연구단) > 1. Journal Papers (저널논문)
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