Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine
Abstract Translational medicine aims to improve human health by exploring potential treatment methods developed during basic scientific research and applying them to the treatment of patients in clinical settings. The advanced perceptions of gene functions have remarkably revolutionized clinical tre...
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2023-09-01
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| Series: | Advanced Science |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/advs.202300195 |
| _version_ | 1797692637288857600 |
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| author | Ruixuan Zheng Lexiang Zhang Rokshana Parvin Lihuang Su Junjie Chi Keqing Shi Fangfu Ye Xiaoying Huang |
| author_facet | Ruixuan Zheng Lexiang Zhang Rokshana Parvin Lihuang Su Junjie Chi Keqing Shi Fangfu Ye Xiaoying Huang |
| author_sort | Ruixuan Zheng |
| collection | DOAJ |
| description | Abstract Translational medicine aims to improve human health by exploring potential treatment methods developed during basic scientific research and applying them to the treatment of patients in clinical settings. The advanced perceptions of gene functions have remarkably revolutionized clinical treatment strategies for target agents. However, the progress in gene editing therapy has been hindered due to the severe off‐target effects and limited editing sites. Fortunately, the development in the clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR‐Cas9) system has renewed hope for gene therapy field. The CRISPR‐Cas9 system can fulfill various simple or complex purposes, including gene knockout, knock‐in, activation, interference, base editing, and sequence detection. Accordingly, the CRISPR‐Cas9 system is adaptable to translational medicine, which calls for the alteration of genomic sequences. This review aims to present the latest CRISPR‐Cas9 technology achievements and prospect to translational medicine advances. The principle and characterization of the CRISPR‐Cas9 system are firstly introduced. The authors then focus on recent pre‐clinical and clinical research directions, including the construction of disease models, disease‐related gene screening and regulation, and disease treatment and diagnosis for multiple refractory diseases. Finally, some clinical challenges including off‐target effects, in vivo vectors, and ethical problems, and future perspective are also discussed. |
| first_indexed | 2024-03-12T02:31:28Z |
| format | Article |
| id | doaj.art-006db6972b204b93b656c4db619c00aa |
| institution | Directory Open Access Journal |
| issn | 2198-3844 |
| language | English |
| last_indexed | 2024-03-12T02:31:28Z |
| publishDate | 2023-09-01 |
| publisher | Wiley |
| record_format | Article |
| series | Advanced Science |
| spelling | doaj.art-006db6972b204b93b656c4db619c00aa2023-09-05T07:49:09ZengWileyAdvanced Science2198-38442023-09-011025n/an/a10.1002/advs.202300195Progress and Perspective of CRISPR‐Cas9 Technology in Translational MedicineRuixuan Zheng0Lexiang Zhang1Rokshana Parvin2Lihuang Su3Junjie Chi4Keqing Shi5Fangfu Ye6Xiaoying Huang7Joint Centre of Translational Medicine The First Affiliated Hospital of Wenzhou Medical University WenzhouZhejiang 325000P. R. ChinaJoint Centre of Translational Medicine The First Affiliated Hospital of Wenzhou Medical University WenzhouZhejiang 325000P. R. ChinaOujiang Laboratory (Zhejiang Lab for Regenerative Medicine Vision and Brain Health); Wenzhou Institute University of Chinese Academy of Sciences WenzhouZhejiang 325000P. R. ChinaJoint Centre of Translational Medicine The First Affiliated Hospital of Wenzhou Medical University WenzhouZhejiang 325000P. R. ChinaJoint Centre of Translational Medicine The First Affiliated Hospital of Wenzhou Medical University WenzhouZhejiang 325000P. R. ChinaJoint Centre of Translational Medicine The First Affiliated Hospital of Wenzhou Medical University WenzhouZhejiang 325000P. R. ChinaJoint Centre of Translational Medicine The First Affiliated Hospital of Wenzhou Medical University WenzhouZhejiang 325000P. R. ChinaJoint Centre of Translational Medicine The First Affiliated Hospital of Wenzhou Medical University WenzhouZhejiang 325000P. R. ChinaAbstract Translational medicine aims to improve human health by exploring potential treatment methods developed during basic scientific research and applying them to the treatment of patients in clinical settings. The advanced perceptions of gene functions have remarkably revolutionized clinical treatment strategies for target agents. However, the progress in gene editing therapy has been hindered due to the severe off‐target effects and limited editing sites. Fortunately, the development in the clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR‐Cas9) system has renewed hope for gene therapy field. The CRISPR‐Cas9 system can fulfill various simple or complex purposes, including gene knockout, knock‐in, activation, interference, base editing, and sequence detection. Accordingly, the CRISPR‐Cas9 system is adaptable to translational medicine, which calls for the alteration of genomic sequences. This review aims to present the latest CRISPR‐Cas9 technology achievements and prospect to translational medicine advances. The principle and characterization of the CRISPR‐Cas9 system are firstly introduced. The authors then focus on recent pre‐clinical and clinical research directions, including the construction of disease models, disease‐related gene screening and regulation, and disease treatment and diagnosis for multiple refractory diseases. Finally, some clinical challenges including off‐target effects, in vivo vectors, and ethical problems, and future perspective are also discussed.https://doi.org/10.1002/advs.202300195clustered regularly interspaced short palindromic repeats‐associated protein 9 (CRISPR‐Cas9)gene regulationgene therapytranslational medicine |
| spellingShingle | Ruixuan Zheng Lexiang Zhang Rokshana Parvin Lihuang Su Junjie Chi Keqing Shi Fangfu Ye Xiaoying Huang Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine Advanced Science clustered regularly interspaced short palindromic repeats‐associated protein 9 (CRISPR‐Cas9) gene regulation gene therapy translational medicine |
| title | Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine |
| title_full | Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine |
| title_fullStr | Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine |
| title_full_unstemmed | Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine |
| title_short | Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine |
| title_sort | progress and perspective of crispr cas9 technology in translational medicine |
| topic | clustered regularly interspaced short palindromic repeats‐associated protein 9 (CRISPR‐Cas9) gene regulation gene therapy translational medicine |
| url | https://doi.org/10.1002/advs.202300195 |
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