Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration
Undesired chromosomal translocations, vector integrations, and large deletions remain a problem for therapeutic gene editing in vivo. Here, the authors compare the CRISPR-Cas9TX variant with CRISPR-Cas9 and show elimination of chromosomal translocations and reduction of AVV integration when targetin...
| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2022-12-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-022-35640-4 |
| _version_ | 1797977441499611136 |
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| author | Jianhang Yin Kailun Fang Yanxia Gao Liqiong Ou Shaopeng Yuan Changchang Xin Weiwei Wu Wei-wei Wu Jiaxu Hong Hui Yang Jiazhi Hu |
| author_facet | Jianhang Yin Kailun Fang Yanxia Gao Liqiong Ou Shaopeng Yuan Changchang Xin Weiwei Wu Wei-wei Wu Jiaxu Hong Hui Yang Jiazhi Hu |
| author_sort | Jianhang Yin |
| collection | DOAJ |
| description | Undesired chromosomal translocations, vector integrations, and large deletions remain a problem for therapeutic gene editing in vivo. Here, the authors compare the CRISPR-Cas9TX variant with CRISPR-Cas9 and show elimination of chromosomal translocations and reduction of AVV integration when targeting Vegfa for the treatment of age-related macular degeneration in a mouse model. |
| first_indexed | 2024-04-11T05:06:58Z |
| format | Article |
| id | doaj.art-ead978410b604a268f507033d862566f |
| institution | Directory Open Access Journal |
| issn | 2041-1723 |
| language | English |
| last_indexed | 2024-04-11T05:06:58Z |
| publishDate | 2022-12-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj.art-ead978410b604a268f507033d862566f2022-12-25T12:20:53ZengNature PortfolioNature Communications2041-17232022-12-011311810.1038/s41467-022-35640-4Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degenerationJianhang Yin0Kailun Fang1Yanxia Gao2Liqiong Ou3Shaopeng Yuan4Changchang Xin5Weiwei Wu6Wei-wei Wu7Jiaxu Hong8Hui Yang9Jiazhi Hu10The MOE Key Laboratory of Cell Proliferation and Differentiation, School of Life Sciences, Center for Life Sciences, Genome Editing Research Center, Peking UniversityInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of SciencesInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of SciencesThe MOE Key Laboratory of Cell Proliferation and Differentiation, School of Life Sciences, Center for Life Sciences, Genome Editing Research Center, Peking UniversityThe MOE Key Laboratory of Cell Proliferation and Differentiation, School of Life Sciences, Center for Life Sciences, Genome Editing Research Center, Peking UniversityThe MOE Key Laboratory of Cell Proliferation and Differentiation, School of Life Sciences, Center for Life Sciences, Genome Editing Research Center, Peking UniversityInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of SciencesInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of SciencesDepartment of Ophthalmology and Vision Science, Shanghai Eye, Ear, Nose and Throat Hospital, Fudan UniversityInstitute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Research Center for Brain Science and Brain-Inspired Intelligence, Shanghai Institutes for Biological Sciences, Chinese Academy of SciencesThe MOE Key Laboratory of Cell Proliferation and Differentiation, School of Life Sciences, Center for Life Sciences, Genome Editing Research Center, Peking UniversityUndesired chromosomal translocations, vector integrations, and large deletions remain a problem for therapeutic gene editing in vivo. Here, the authors compare the CRISPR-Cas9TX variant with CRISPR-Cas9 and show elimination of chromosomal translocations and reduction of AVV integration when targeting Vegfa for the treatment of age-related macular degeneration in a mouse model.https://doi.org/10.1038/s41467-022-35640-4 |
| spellingShingle | Jianhang Yin Kailun Fang Yanxia Gao Liqiong Ou Shaopeng Yuan Changchang Xin Weiwei Wu Wei-wei Wu Jiaxu Hong Hui Yang Jiazhi Hu Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration Nature Communications |
| title | Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration |
| title_full | Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration |
| title_fullStr | Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration |
| title_full_unstemmed | Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration |
| title_short | Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration |
| title_sort | safeguarding genome integrity during gene editing therapy in a mouse model of age related macular degeneration |
| url | https://doi.org/10.1038/s41467-022-35640-4 |
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