Precise genome-editing in human diseases: mechanisms, strategies and applications
Abstract Precise genome-editing platforms are versatile tools for generating specific, site-directed DNA insertions, deletions, and substitutions. The continuous enhancement of these tools has led to a revolution in the life sciences, which promises to deliver novel therapies for genetic disease. Pr...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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Nature Publishing Group
2024-02-01
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| Series: | Signal Transduction and Targeted Therapy |
| Online Access: | https://doi.org/10.1038/s41392-024-01750-2 |
| _version_ | 1797273138021531648 |
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| author | Yanjiang Zheng Yifei Li Kaiyu Zhou Tiange Li Nathan J. VanDusen Yimin Hua |
| author_facet | Yanjiang Zheng Yifei Li Kaiyu Zhou Tiange Li Nathan J. VanDusen Yimin Hua |
| author_sort | Yanjiang Zheng |
| collection | DOAJ |
| description | Abstract Precise genome-editing platforms are versatile tools for generating specific, site-directed DNA insertions, deletions, and substitutions. The continuous enhancement of these tools has led to a revolution in the life sciences, which promises to deliver novel therapies for genetic disease. Precise genome-editing can be traced back to the 1950s with the discovery of DNA’s double-helix and, after 70 years of development, has evolved from crude in vitro applications to a wide range of sophisticated capabilities, including in vivo applications. Nonetheless, precise genome-editing faces constraints such as modest efficiency, delivery challenges, and off-target effects. In this review, we explore precise genome-editing, with a focus on introduction of the landmark events in its history, various platforms, delivery systems, and applications. First, we discuss the landmark events in the history of precise genome-editing. Second, we describe the current state of precise genome-editing strategies and explain how these techniques offer unprecedented precision and versatility for modifying the human genome. Third, we introduce the current delivery systems used to deploy precise genome-editing components through DNA, RNA, and RNPs. Finally, we summarize the current applications of precise genome-editing in labeling endogenous genes, screening genetic variants, molecular recording, generating disease models, and gene therapy, including ex vivo therapy and in vivo therapy, and discuss potential future advances. |
| first_indexed | 2024-03-07T14:39:18Z |
| format | Article |
| id | doaj.art-f01727281bdf400987561e50174e7243 |
| institution | Directory Open Access Journal |
| issn | 2059-3635 |
| language | English |
| last_indexed | 2024-03-07T14:39:18Z |
| publishDate | 2024-02-01 |
| publisher | Nature Publishing Group |
| record_format | Article |
| series | Signal Transduction and Targeted Therapy |
| spelling | doaj.art-f01727281bdf400987561e50174e72432024-03-05T20:29:28ZengNature Publishing GroupSignal Transduction and Targeted Therapy2059-36352024-02-019112110.1038/s41392-024-01750-2Precise genome-editing in human diseases: mechanisms, strategies and applicationsYanjiang Zheng0Yifei Li1Kaiyu Zhou2Tiange Li3Nathan J. VanDusen4Yimin Hua5Key Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, Department of Pediatrics, West China Second University Hospital, Sichuan UniversityKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, Department of Pediatrics, West China Second University Hospital, Sichuan UniversityKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, Department of Pediatrics, West China Second University Hospital, Sichuan UniversityDepartment of Cardiovascular Surgery, West China Hospital, Sichuan UniversityDepartment of Pediatrics, Herman B Wells Center for Pediatric Research, Indiana University School of MedicineKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, Department of Pediatrics, West China Second University Hospital, Sichuan UniversityAbstract Precise genome-editing platforms are versatile tools for generating specific, site-directed DNA insertions, deletions, and substitutions. The continuous enhancement of these tools has led to a revolution in the life sciences, which promises to deliver novel therapies for genetic disease. Precise genome-editing can be traced back to the 1950s with the discovery of DNA’s double-helix and, after 70 years of development, has evolved from crude in vitro applications to a wide range of sophisticated capabilities, including in vivo applications. Nonetheless, precise genome-editing faces constraints such as modest efficiency, delivery challenges, and off-target effects. In this review, we explore precise genome-editing, with a focus on introduction of the landmark events in its history, various platforms, delivery systems, and applications. First, we discuss the landmark events in the history of precise genome-editing. Second, we describe the current state of precise genome-editing strategies and explain how these techniques offer unprecedented precision and versatility for modifying the human genome. Third, we introduce the current delivery systems used to deploy precise genome-editing components through DNA, RNA, and RNPs. Finally, we summarize the current applications of precise genome-editing in labeling endogenous genes, screening genetic variants, molecular recording, generating disease models, and gene therapy, including ex vivo therapy and in vivo therapy, and discuss potential future advances.https://doi.org/10.1038/s41392-024-01750-2 |
| spellingShingle | Yanjiang Zheng Yifei Li Kaiyu Zhou Tiange Li Nathan J. VanDusen Yimin Hua Precise genome-editing in human diseases: mechanisms, strategies and applications Signal Transduction and Targeted Therapy |
| title | Precise genome-editing in human diseases: mechanisms, strategies and applications |
| title_full | Precise genome-editing in human diseases: mechanisms, strategies and applications |
| title_fullStr | Precise genome-editing in human diseases: mechanisms, strategies and applications |
| title_full_unstemmed | Precise genome-editing in human diseases: mechanisms, strategies and applications |
| title_short | Precise genome-editing in human diseases: mechanisms, strategies and applications |
| title_sort | precise genome editing in human diseases mechanisms strategies and applications |
| url | https://doi.org/10.1038/s41392-024-01750-2 |
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