AAV-mediated gene therapies by miniature gene editing tools
文献类型：期刊论文
| 作者 | Kong, Xiangfeng2,3; Li, Tong1,3; Yang, Hui1,2,3,4 |
| 刊名 | SCIENCE CHINA-LIFE SCIENCES
 |
| 出版日期 | 2024-09-27 |
| 页码 | 14 |
| 关键词 | gene therapy adeno-associated viruses (AAVs) genome editing miniature CRISPR-Cas Obligate Mobile Element-Guided Activity (OMEGA) |
| ISSN号 | 1674-7305 |
| DOI | 10.1007/s11427-023-2608-5 |
| 通讯作者 | Kong, Xiangfeng(kongxiangfeng@simm.ac.cn) ; Yang, Hui(huiyang@ion.ac.cn) |
| 英文摘要 | The advent of CRISPR-Cas has revolutionized precise gene editing. While pioneering CRISPR nucleases like Cas9 and Cas12 generate targeted DNA double-strand breaks (DSB) for knockout or homology-directed repair, next generation CRISPR technologies enable gene editing without DNA DSB. Base editors directly convert bases, prime editors make diverse alterations, and dead Cas-regulator fusions allow nuanced control of gene expression, avoiding potentially risks like translocations. Meanwhile, the discovery of diminutive Cas12 orthologs and Obligate Mobile Element-Guided Activity (OMEGA) nucleases has overcome cargo limitations of adeno-associated viral vectors, expanding prospects for in vivo therapeutic delivery. Here, we review the ever-evolving landscape of cutting-edge gene editing tools, focusing on miniature Cas12 orthologs and OMEGA effectors amenable to single AAV packaging. We also summarize CRISPR therapies delivered using AAV vectors, discuss challenges such as efficiency and specificity, and look to the future of this transformative field of in vivo gene editing enabled by AAV vectors delivery. |
| WOS关键词 | NUCLEIC-ACID DETECTION ; IN-VIVO ; STRUCTURAL BASIS ; HYPERTROPHIC CARDIOMYOPATHY ; PACKAGING CAPACITY ; GENOMIC DNA ; CRISPR ; BASE ; VECTOR ; SYSTEM |
| 资助项目 | National Science and Technology Innovation[2021ZD0200900] ; National Natural Science Foundation of China[31925016] ; National Natural Science Foundation of China[82021001] ; Basic Frontier Scientific Research Program of Chinese Academy of Sciences[ZDBS-LY-SM001] ; Project of Shanghai Municipal Science and Technology Commission[20MC1920400] ; HuidaGene Therapeutics Co., Ltd. ; Shanghai Center for Brain Science and Brian-Inspired Technology |
| WOS研究方向 | Life Sciences & Biomedicine - Other Topics |
| 语种 | 英语 |
| WOS记录号 | WOS:001329829200001 |
| 出版者 | SCIENCE PRESS |
| 源URL | [http://119.78.100.183/handle/2S10ELR8/313781]  |
| 专题 | 中国科学院上海药物研究所 |
| 通讯作者 | Kong, Xiangfeng; Yang, Hui |
| 作者单位 | 1.HuidaGene Therapeut Co Ltd, Shanghai 200131, Peoples R China 2.Chinese Acad Sci, Inst Neurosci, Ctr Excellence Brain Sci & Intelligence Technol, Shanghai 200031, Peoples R China 3.Chinese Acad Sci, Shanghai Inst Mat Med, Shanghai 201203, Peoples R China 4.Shanghai Ctr Brain Sci & Brain Inspired Technol, Shanghai 201210, Peoples R China |
| 推荐引用方式 GB/T 7714 | Kong, Xiangfeng,Li, Tong,Yang, Hui. AAV-mediated gene therapies by miniature gene editing tools[J]. SCIENCE CHINA-LIFE SCIENCES,2024:14. |
| APA | Kong, Xiangfeng,Li, Tong,&Yang, Hui.(2024).AAV-mediated gene therapies by miniature gene editing tools.SCIENCE CHINA-LIFE SCIENCES,14. |
| MLA | Kong, Xiangfeng,et al."AAV-mediated gene therapies by miniature gene editing tools".SCIENCE CHINA-LIFE SCIENCES (2024):14. |
入库方式： OAI收割
来源：上海药物研究所
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