Poly-sgRNA/siRNA ribonucleoprotein nanoparticles for targeted gene disruption

Title
Poly-sgRNA/siRNA ribonucleoprotein nanoparticles for targeted gene disruption
Authors
안대로정학숙하종성이재성정재필김혜진변주영김상아이희재이종범
Keywords
poly-ribonucleoprotein; CRISPR/Cas9; rolling circle amplification; gene disruption; gene delivery
Issue Date
2017-03
Publisher
Journal of controlled release
Citation
VOL 250-35
Abstract
Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein-9 nuclease (Cas9) can be used for the specific disruption of a target gene to permanently suppress the expression of the protein encoded by the target gene. Efficient delivery of the system to an intracellular target site should be achieved to utilize the tremendous potential of the genome-editing tool in biomedical applications such as the knock-out of disease-related genes and the correction of defect genes. Here, we devise polymeric CRISPR/Cas9 system based on poly-ribonucleoprotein (RNP) nanoparticles consisting of polymeric sgRNA, siRNA, and Cas9 endonuclease in order to improve the delivery efficiency. When delivered by cationic lipids, the RNP nanoparticles built with chimeric poly-sgRNA/siRNA sequences generate multiple sgRNA-Cas9 RNP complexes upon the Dicer-mediated digestion of the siRNA parts, leading to more efficient disruption of the target gene in cells and animal models, compared with the monomeric sgRNA-Cas9 RNP complex.
URI
http://pubs.kist.re.kr/handle/201004/66585
ISSN
0168-3659
Appears in Collections:
KIST Publication > Article
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