Poly-sgRNA/siRNA ribonucleoprotein nanoparticles for targeted gene disruption
- Poly-sgRNA/siRNA ribonucleoprotein nanoparticles for targeted gene disruption
- 안대로; 정학숙; 하종성; 이재성; 정재필; 김혜진; 변주영; 김상아; 이희재; 이종범
- poly-ribonucleoprotein; CRISPR/Cas9; rolling circle amplification; gene disruption; gene delivery
- Issue Date
- Journal of controlled release
- VOL 250-35
- 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.
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