DataVXSmallPaper
journal article·2019-11

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

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

Small· Volume 15· Issue 46
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Source abstract

Abstract

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 ≈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.
Source: crossref · Quality: baseline

Research context

NanomaterialsBiomaterials

OpenAlex topics: CRISPR and Genetic Engineering · RNA Interference and Gene Delivery · Advanced biosensing and bioanalysis techniques

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

SKKU Advanced Institute of Nanotechnology (SAINT) Sungkyunkwan University South Suwon 16419 Korea

Department of Biomedical Engineering Sungkyunkwan University Suwon 16419 South Korea

Center for Neuroscience Imaging Research (CNIR) Institute for Basic Science (IBS) Sungkyunkwan University Suwon 16419 South Korea

Department of Medicine Graduate School Kyung Hee University Seoul 02447 South Korea

Department of Nano Engineering Sungkyunkwan University Suwon 16419 South Korea

Department of Pathology College of Medicine Kyung Hee University Seoul 02447 South Korea

Department of Biomedical Science Graduate School Kyung Hee University Seoul 02447 South Korea

Biomedical Institute for Convergence at SKKU (BICS) Sungkyunkwan University Suwon 16419 South Korea

Funding

Institute for Basic Science · Korea Institute of Science and Technology · National Research Foundation of Korea · Ministry of Education

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