Advancements in CRISPR/Cas systems for disease treatment
The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas (CRISPR-associated proteins) is an adaptive immune system present in most bacteria and archaea, protecting them from infection by exogenous genetic elements. Due to its simplicity, cost-effectiveness, and precise gene editing...
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| Format: | Article |
| Language: | English |
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Elsevier
2025-06-01
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| Series: | Acta Pharmaceutica Sinica B |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2211383525003132 |
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| author | Yangsong Xu Hao Le Qinjie Wu Ning Wang Changyang Gong |
| author_facet | Yangsong Xu Hao Le Qinjie Wu Ning Wang Changyang Gong |
| author_sort | Yangsong Xu |
| collection | DOAJ |
| description | The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas (CRISPR-associated proteins) is an adaptive immune system present in most bacteria and archaea, protecting them from infection by exogenous genetic elements. Due to its simplicity, cost-effectiveness, and precise gene editing capabilities, CRISPR/Cas technology has emerged as a promising tool for treating diseases. The continuous refinement of derivative systems has further broadened its scope in disease treatment. Nevertheless, the heterogeneous physiopathological nature of diseases and variations in disease onset sites pose significant challenges for in vivo applications of CRISPR systems. The efficiency of CRISPR systems in disease treatment is directly influenced by the performance of the delivery system. Additionally, concerns such as off-target effects present crucial hurdles in the clinical implementation of CRISPR systems. This review provides a comprehensive overview of the development of CRISPR systems, vector technologies, and their applications in disease treatment, while also addressing the challenges encountered in clinical settings. Furthermore, future research directions are outlined to pave the way for advancements in CRISPR-based therapies. |
| format | Article |
| id | doaj-art-d1ec8967a85447b8b9f8de4d7de275af |
| institution | OA Journals |
| issn | 2211-3835 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Acta Pharmaceutica Sinica B |
| spelling | doaj-art-d1ec8967a85447b8b9f8de4d7de275af2025-08-20T02:22:01ZengElsevierActa Pharmaceutica Sinica B2211-38352025-06-011562818284410.1016/j.apsb.2025.05.007Advancements in CRISPR/Cas systems for disease treatmentYangsong Xu0Hao Le1Qinjie Wu2Ning Wang3Changyang Gong4Department of Biotherapy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaDepartment of Biotherapy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaDepartment of Biotherapy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaCorresponding authors.; Department of Biotherapy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaCorresponding authors.; Department of Biotherapy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaThe clustered regularly interspaced short palindromic repeats (CRISPR)-Cas (CRISPR-associated proteins) is an adaptive immune system present in most bacteria and archaea, protecting them from infection by exogenous genetic elements. Due to its simplicity, cost-effectiveness, and precise gene editing capabilities, CRISPR/Cas technology has emerged as a promising tool for treating diseases. The continuous refinement of derivative systems has further broadened its scope in disease treatment. Nevertheless, the heterogeneous physiopathological nature of diseases and variations in disease onset sites pose significant challenges for in vivo applications of CRISPR systems. The efficiency of CRISPR systems in disease treatment is directly influenced by the performance of the delivery system. Additionally, concerns such as off-target effects present crucial hurdles in the clinical implementation of CRISPR systems. This review provides a comprehensive overview of the development of CRISPR systems, vector technologies, and their applications in disease treatment, while also addressing the challenges encountered in clinical settings. Furthermore, future research directions are outlined to pave the way for advancements in CRISPR-based therapies.http://www.sciencedirect.com/science/article/pii/S2211383525003132CRISPR/Cas9CRISPR/Cas classificationGene editingDisease treatmentDeliver |
| spellingShingle | Yangsong Xu Hao Le Qinjie Wu Ning Wang Changyang Gong Advancements in CRISPR/Cas systems for disease treatment Acta Pharmaceutica Sinica B CRISPR/Cas9 CRISPR/Cas classification Gene editing Disease treatment Deliver |
| title | Advancements in CRISPR/Cas systems for disease treatment |
| title_full | Advancements in CRISPR/Cas systems for disease treatment |
| title_fullStr | Advancements in CRISPR/Cas systems for disease treatment |
| title_full_unstemmed | Advancements in CRISPR/Cas systems for disease treatment |
| title_short | Advancements in CRISPR/Cas systems for disease treatment |
| title_sort | advancements in crispr cas systems for disease treatment |
| topic | CRISPR/Cas9 CRISPR/Cas classification Gene editing Disease treatment Deliver |
| url | http://www.sciencedirect.com/science/article/pii/S2211383525003132 |
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