期刊
SMALL
卷 15, 期 46, 页码 -出版社
WILEY-V C H VERLAG GMBH
DOI: 10.1002/smll.201903172
关键词
delivery; dNP2 lipopeptides; gene editing; hyper-accurate Cas9; nanosomes
类别
资金
- Institute for Basic Science [IBS-R015-D1]
- Korea Institute of Science and Technology [KIST-2E29563-19-119]
- National Research Foundation of Korea (NRF) - Ministry of Education [NRF-2017R1A1B0306021]
- IMNEWRUN CORPORATION
A transient cytosolic delivery system for accurate Cas9 ribonucleoprotein is a key factor for target specificity of the CRIPSR/Cas9 toolkit. Owing to the large size of the Cas9 protein and a long negative strand RNA, the development of the delivery system is still a major challenge. Here, a size-controlled lipopeptide-based nanosome system is reported, derived from the blood-brain barrier-permeable dNP2 peptide which is capable of delivering a hyperaccurate Cas9 ribonucleoprotein complex (HypaRNP) into human cells for gene editing. Each nanosome is capable of encapsulating and delivering approximate to 2 HypaRNP molecules into the cytoplasm, followed by nuclear localization at 4 h post-treatment without significant cytotoxicity. The HypaRNP thus efficiently enacts endogenous eGFP silencing and editing in human embryonic kidney cells (up to 27.6%) and glioblastoma (up to 19.7% frequency of modification). The lipopeptide-based nanosome system shows superior delivery efficiency, high controllability, and simplicity, thus providing biocompatibility and versatile platform approach for CRISPR-mediated transient gene editing applications.
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