From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine
Abstract CRISPR-based gene editing technology theoretically allows for precise manipulation of any genetic target within living cells, achieving the desired sequence modifications. This revolutionary advancement has fundamentally transformed the field of biomedicine, offering immense clinical potent...
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| Format: | Article |
| Language: | English |
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BMC
2024-12-01
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| Series: | Journal of Translational Medicine |
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| Online Access: | https://doi.org/10.1186/s12967-024-05957-3 |
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| author | Weihui Xu Shiyao Zhang Huan Qin Kai Yao |
| author_facet | Weihui Xu Shiyao Zhang Huan Qin Kai Yao |
| author_sort | Weihui Xu |
| collection | DOAJ |
| description | Abstract CRISPR-based gene editing technology theoretically allows for precise manipulation of any genetic target within living cells, achieving the desired sequence modifications. This revolutionary advancement has fundamentally transformed the field of biomedicine, offering immense clinical potential for treating and correcting genetic disorders. In the treatment of most genetic diseases, precise genome editing that avoids the generation of mixed editing byproducts is considered the ideal approach. This article reviews the current progress of base editors and prime editors, elaborating on specific examples of their applications in the therapeutic field, and highlights opportunities for improvement. Furthermore, we discuss the specific performance of these technologies in terms of safety and efficacy in clinical applications, and analyze the latest advancements and potential directions that could influence the future development of genome editing technologies. Our goal is to outline the clinical relevance of this rapidly evolving scientific field and preview a roadmap for successful DNA base editing therapies for the treatment of hereditary or idiopathic diseases. |
| format | Article |
| id | doaj-art-caaec67d6e8f4cc8b18e028a61dddf62 |
| institution | OA Journals |
| issn | 1479-5876 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | BMC |
| record_format | Article |
| series | Journal of Translational Medicine |
| spelling | doaj-art-caaec67d6e8f4cc8b18e028a61dddf622025-08-20T01:57:12ZengBMCJournal of Translational Medicine1479-58762024-12-0122114310.1186/s12967-024-05957-3From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicineWeihui Xu0Shiyao Zhang1Huan Qin2Kai Yao3Institute of Visual Neuroscience and Stem Cell Engineering, Wuhan University of Science and TechnologyInstitute of Visual Neuroscience and Stem Cell Engineering, Wuhan University of Science and TechnologyInstitute of Visual Neuroscience and Stem Cell Engineering, Wuhan University of Science and TechnologyInstitute of Visual Neuroscience and Stem Cell Engineering, Wuhan University of Science and TechnologyAbstract CRISPR-based gene editing technology theoretically allows for precise manipulation of any genetic target within living cells, achieving the desired sequence modifications. This revolutionary advancement has fundamentally transformed the field of biomedicine, offering immense clinical potential for treating and correcting genetic disorders. In the treatment of most genetic diseases, precise genome editing that avoids the generation of mixed editing byproducts is considered the ideal approach. This article reviews the current progress of base editors and prime editors, elaborating on specific examples of their applications in the therapeutic field, and highlights opportunities for improvement. Furthermore, we discuss the specific performance of these technologies in terms of safety and efficacy in clinical applications, and analyze the latest advancements and potential directions that could influence the future development of genome editing technologies. Our goal is to outline the clinical relevance of this rapidly evolving scientific field and preview a roadmap for successful DNA base editing therapies for the treatment of hereditary or idiopathic diseases.https://doi.org/10.1186/s12967-024-05957-3CRISPR–CasGenome editingBase editingPrime editingGene therapy deliveryClinical treatment |
| spellingShingle | Weihui Xu Shiyao Zhang Huan Qin Kai Yao From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine Journal of Translational Medicine CRISPR–Cas Genome editing Base editing Prime editing Gene therapy delivery Clinical treatment |
| title | From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine |
| title_full | From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine |
| title_fullStr | From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine |
| title_full_unstemmed | From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine |
| title_short | From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine |
| title_sort | from bench to bedside cutting edge applications of base editing and prime editing in precision medicine |
| topic | CRISPR–Cas Genome editing Base editing Prime editing Gene therapy delivery Clinical treatment |
| url | https://doi.org/10.1186/s12967-024-05957-3 |
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