Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery
Genome editing: Novel editors for CRISPR-Cas9 technology Tiny genome editors that can fit inside a single delivery system could potentially improve the efficiency and specificity of the editing technology known as CRISPR-Cas9. This technology is used to edit parts of the genome and shows promise for...
| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Publishing Group
2023-02-01
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| Series: | Experimental and Molecular Medicine |
| Online Access: | https://doi.org/10.1038/s12276-023-00938-w |
| _version_ | 1797865471257608192 |
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| author | Jiyeon Kweon An-Hee Jang Eunji Kwon Ungi Kim Ha Rim Shin Jieun See Gayoung Jang Chaeyeon Lee Taeyoung Koo Seokjoong Kim Yongsub Kim |
| author_facet | Jiyeon Kweon An-Hee Jang Eunji Kwon Ungi Kim Ha Rim Shin Jieun See Gayoung Jang Chaeyeon Lee Taeyoung Koo Seokjoong Kim Yongsub Kim |
| author_sort | Jiyeon Kweon |
| collection | DOAJ |
| description | Genome editing: Novel editors for CRISPR-Cas9 technology Tiny genome editors that can fit inside a single delivery system could potentially improve the efficiency and specificity of the editing technology known as CRISPR-Cas9. This technology is used to edit parts of the genome and shows promise for disease therapeutics. Cas9 is a bacterial enzyme that acts as molecular scissors to edit DNA and is guided to the correct DNA region by specific RNA segments (gRNAs). Yongsub Kim at the University of Ulsan in Seoul, South Korea, and co-workers used one of the smallest known Cas9 enzymes, sourced from Campylobactor jejuni, to create two ‘base editors’ (BEs), capable of specific base substitutions. Results suggest the BEs are highly efficient, covering more potential targets than previous Cas9-based BEs. One of the BEs can be packaged, alongside two gRNAs, into a single viral vector for targeted delivery. |
| first_indexed | 2024-04-09T23:09:02Z |
| format | Article |
| id | doaj.art-5a3318fd8f0248789017a3d9b04bef91 |
| institution | Directory Open Access Journal |
| issn | 2092-6413 |
| language | English |
| last_indexed | 2024-04-09T23:09:02Z |
| publishDate | 2023-02-01 |
| publisher | Nature Publishing Group |
| record_format | Article |
| series | Experimental and Molecular Medicine |
| spelling | doaj.art-5a3318fd8f0248789017a3d9b04bef912023-03-22T10:31:26ZengNature Publishing GroupExperimental and Molecular Medicine2092-64132023-02-0155237738410.1038/s12276-023-00938-wTargeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated deliveryJiyeon Kweon0An-Hee Jang1Eunji Kwon2Ungi Kim3Ha Rim Shin4Jieun See5Gayoung Jang6Chaeyeon Lee7Taeyoung Koo8Seokjoong Kim9Yongsub Kim10Department of Biomedical Sciences, Asan Medical Institute of Convergence Science and Technology, Asan Medical Center, University of Ulsan College of MedicineDepartment of Biomedical Sciences, Asan Medical Institute of Convergence Science and Technology, Asan Medical Center, University of Ulsan College of MedicineDepartment of Fundamental Pharmaceutical Sciences, Kyung Hee UniversityToolgen, Inc.Department of Biomedical Sciences, Asan Medical Institute of Convergence Science and Technology, Asan Medical Center, University of Ulsan College of MedicineDepartment of Biomedical Sciences, Asan Medical Institute of Convergence Science and Technology, Asan Medical Center, University of Ulsan College of MedicineDepartment of Biomedical Sciences, Asan Medical Institute of Convergence Science and Technology, Asan Medical Center, University of Ulsan College of MedicineDepartment of Biomedical Sciences, Asan Medical Institute of Convergence Science and Technology, Asan Medical Center, University of Ulsan College of MedicineDepartment of Fundamental Pharmaceutical Sciences, Kyung Hee UniversityToolgen, Inc.Department of Biomedical Sciences, Asan Medical Institute of Convergence Science and Technology, Asan Medical Center, University of Ulsan College of MedicineGenome editing: Novel editors for CRISPR-Cas9 technology Tiny genome editors that can fit inside a single delivery system could potentially improve the efficiency and specificity of the editing technology known as CRISPR-Cas9. This technology is used to edit parts of the genome and shows promise for disease therapeutics. Cas9 is a bacterial enzyme that acts as molecular scissors to edit DNA and is guided to the correct DNA region by specific RNA segments (gRNAs). Yongsub Kim at the University of Ulsan in Seoul, South Korea, and co-workers used one of the smallest known Cas9 enzymes, sourced from Campylobactor jejuni, to create two ‘base editors’ (BEs), capable of specific base substitutions. Results suggest the BEs are highly efficient, covering more potential targets than previous Cas9-based BEs. One of the BEs can be packaged, alongside two gRNAs, into a single viral vector for targeted delivery.https://doi.org/10.1038/s12276-023-00938-w |
| spellingShingle | Jiyeon Kweon An-Hee Jang Eunji Kwon Ungi Kim Ha Rim Shin Jieun See Gayoung Jang Chaeyeon Lee Taeyoung Koo Seokjoong Kim Yongsub Kim Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery Experimental and Molecular Medicine |
| title | Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery |
| title_full | Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery |
| title_fullStr | Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery |
| title_full_unstemmed | Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery |
| title_short | Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery |
| title_sort | targeted dual base editing with campylobacter jejuni cas9 by single aav mediated delivery |
| url | https://doi.org/10.1038/s12276-023-00938-w |
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