[논문]Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy

Ryu, Nari; Kim, Min-A; Park, Dongsik; Lee, Byeonghyeon; Kim, Ye-Ri; Kim, Kyung-Hee; Baek, Jeong-In; Kim, Won Jong; Lee, Kyu-Yup; Kim, Un-Kyung

doi:10.1016/j.nano.2018.06.009

[해외논문] Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy

Nanomedicine : nanotechnology, biology and medicine, v.14 no.7, 2018년, pp.2095 - 2102

Ryu, Nari (Department of Biology, College of Natural Sciences, Kyungpook National University, Daegu, Republic of Korea) , Kim, Min-A (Department of Biology, College of Natural Sciences, Kyungpook National University, Daegu, Republic of Korea) , Park, Dongsik (Department of Chemistry, Pohang University of Science and Technology (POSTECH), Pohang, Republic of Korea) , Lee, Byeonghyeon (Department of Biology, College of Natural Sciences, Kyungpook National University, Daegu, Republic of Korea) , Kim, Ye-Ri (Department of Biology, College of Natural Sciences, Kyungpook National University, Daegu, Republic of Korea) , Kim, Kyung-Hee (Department of Biology, College of Natural Sciences, Kyungpook National University, Daegu, Republic of Korea) , Baek, Jeong-In (Department of Aroma-applied Industry, Daegu Haany University, Gyeongsan, Republic of Korea) , Kim, Won Jong (Department of Chemistry, Pohang University of Science and Technology (POSTECH), Pohang, Republic of Korea) , Lee, Kyu-Yup (Department of Otorhinolaryngology-Head and Neck Surgery, School of Medicine, Kyungpook National University, Daegu, Republic of Korea) , Kim, Un-Kyung (Depart)

Abstract ▼ AI-Helper

The-state-of-art CRISPR/Cas9 is one of the most powerful among the approaches being developed to rescue fundamental causes of gene-based inheritable diseases. Several strategies for delivering such genome editing materials have been developed, but the safety, efficacy over time, cost of production, and gene size limitations are still under debate and must be addressed to further improve applications. In this study, we evaluated branched forms of the polyethylenimine (PEI) - branched PEI 25 kDa (BPEI-25K) - and found that it could efficiently deliver CRISPR/Cas9 plasmids. Plasmid DNA expressing both guide RNA and Cas9 to target the Slc26a4 locus was successfully delivered into Neuro2a cells and meditated genome editing within the targeted locus. Our results demonstrated that BPEI-25K is a promising non-viral vector to deliver the CRISPR/Cas9 system in vitro to mediate targeted gene therapy, and these findings contribute to an understanding of CRISPR/Cas9 delivery that may enable development of successful in vivo techniques. (c) 2018 Elsevier Inc. All rights reserved.

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[해외논문] Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy

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[해외논문] Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy

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