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J. Nanobiotechnol., Apr 12 2024, 22 (1) 175 | https://doi.org/10.1186/s12951-024-02427-2 Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing

San Hae Im ^1†, Mincheol Jang ^2†, Ji-Ho Park ^2,3* and Hyun Jung Chung ^1*

¹Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), 291, Daehak-ro, Yuseong-gu, Daejeon 34141, Republic of Korea
²Department of Bio and Brain Engineering, Korea Advanced Institute of Science and Technology (KAIST), 291, Daehak-ro, Yuseong-gu, Daejeon 34141, Republic of Korea
³KAIST Institute for Health Science and Technology, Korea Advanced Institute of Science and Technology (KAIST), 291, Daehak-ro, Yuseong-gu, Daejeon 34141, Republic of Korea

^†San Hae Im and Mincheol Jang contributed equally to this work.
^*Corresponding authors: correspondence to Ji-Ho Park or Hyun Jung Chung

Abstract

Nonviral delivery of the CRISPR/Cas9 system provides great benefits for in vivo gene therapy due to the low risk of side effects. However, in vivo gene editing by delivering the Cas9 ribonucleoprotein (RNP) is challenging due to the poor delivery into target tissues and cells. Here, we introduce an effective delivery method for the CRISPR/Cas9 RNPs by finely tuning the formulation of ionizable lipid nanoparticles. The LNPs delivering CRISPR/Cas9 RNPs (CrLNPs) are demonstrated to induce gene editing with high efficiencies in various cancer cell lines in vitro. Furthermore, we show that CrLNPs can be delivered into tumor tissues with high efficiency, as well as induce significant gene editing in vivo. The current study presents an effective platform for nonviral delivery of the CRISPR/Cas9 system that can be applied as an in vivo gene editing therapeutic for treating various diseases such as cancer and genetic disorders.

논문정보

형식Research article
게재일2024년 04월 (BRIC 등록일 2024-04-15)
연구진국내 연구진
분야 바이오·의료융합 > 바이오센싱 및 나노바이오물질

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