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Lipopeptide-Based Nanosome-Mediated Delivery of Hyperaccurate CRISPR/Cas9 Ribonucleoprotein for Gene Editing

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Title
Lipopeptide-Based Nanosome-Mediated Delivery of Hyperaccurate CRISPR/Cas9 Ribonucleoprotein for Gene Editing
Author(s)
Trung Thanh Thach; Do Hyun Bae; Nam Hyeong Kim; Eun Sung Kang; Bok Soo Lee; Kayoung Han; Minsuk Kwak; Hojae Choi; JiYoung Nam; Taegeun Bae; Minah Suh; Junho K. Hur; Yong Ho Kim
Publication Date
2019-11
Journal
SMALL, v.15, no.46, pp.1903172 -
Publisher
WILEY-V C H VERLAG GMBH
Abstract
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. © 2019 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim
URI
https://pr.ibs.re.kr/handle/8788114/6806
ISSN
1613-6810
Appears in Collections:
Center for Neuroscience Imaging Research (뇌과학 이미징 연구단) > Journal Papers (저널논문)
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