Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing
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 intr...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
BMC
2024-04-01
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| Series: | Journal of Nanobiotechnology |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12951-024-02427-2 |
| _version_ | 1797209037721305088 |
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| author | San Hae Im Mincheol Jang Ji-Ho Park Hyun Jung Chung |
| author_facet | San Hae Im Mincheol Jang Ji-Ho Park Hyun Jung Chung |
| author_sort | San Hae Im |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-24T09:48:20Z |
| format | Article |
| id | doaj.art-08a2eb3f8bee4be89902f7affbdf9232 |
| institution | Directory Open Access Journal |
| issn | 1477-3155 |
| language | English |
| last_indexed | 2024-04-24T09:48:20Z |
| publishDate | 2024-04-01 |
| publisher | BMC |
| record_format | Article |
| series | Journal of Nanobiotechnology |
| spelling | doaj.art-08a2eb3f8bee4be89902f7affbdf92322024-04-14T11:28:21ZengBMCJournal of Nanobiotechnology1477-31552024-04-0122111310.1186/s12951-024-02427-2Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editingSan Hae Im0Mincheol Jang1Ji-Ho Park2Hyun Jung Chung3Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST)Department of Bio and Brain Engineering, Korea Advanced Institute of Science and Technology (KAIST)Department of Bio and Brain Engineering, Korea Advanced Institute of Science and Technology (KAIST)Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST)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.https://doi.org/10.1186/s12951-024-02427-2CRISPR/Cas9Lipid nanoparticlesRibonucleoproteinIn vivo delivery |
| spellingShingle | San Hae Im Mincheol Jang Ji-Ho Park Hyun Jung Chung Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing Journal of Nanobiotechnology CRISPR/Cas9 Lipid nanoparticles Ribonucleoprotein In vivo delivery |
| title | Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing |
| title_full | Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing |
| title_fullStr | Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing |
| title_full_unstemmed | Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing |
| title_short | Finely tuned ionizable lipid nanoparticles for CRISPR/Cas9 ribonucleoprotein delivery and gene editing |
| title_sort | finely tuned ionizable lipid nanoparticles for crispr cas9 ribonucleoprotein delivery and gene editing |
| topic | CRISPR/Cas9 Lipid nanoparticles Ribonucleoprotein In vivo delivery |
| url | https://doi.org/10.1186/s12951-024-02427-2 |
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