Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy
Genome editing technology is a technique for targeted genetic modifications, enabling the knockout and addition of specific DNA fragments. This technology has been widely used in various types of biomedical research, clinics and agriculture. In terms of disease research, constructing appropriate ani...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2019-01-01
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| Series: | Computational and Structural Biotechnology Journal |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2001037019300650 |
| _version_ | 1819016090756317184 |
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| author | Qian Li Zhou Qin Qingnan Wang Ting Xu Yang Yang Zhiyao He |
| author_facet | Qian Li Zhou Qin Qingnan Wang Ting Xu Yang Yang Zhiyao He |
| author_sort | Qian Li |
| collection | DOAJ |
| description | Genome editing technology is a technique for targeted genetic modifications, enabling the knockout and addition of specific DNA fragments. This technology has been widely used in various types of biomedical research, clinics and agriculture. In terms of disease research, constructing appropriate animal models is necessary. Combining reproductive technology with genome editing, many animal disease models have been generated for basic and clinical research. In addition, precisely targeted modifications allow genome editing to flourish in the field of gene therapy. Many mutations refractory to traditional gene therapy could be permanently corrected at the DNA level. Thus, genome editing is undoubtedly a promising technology for gene therapy. In this review, we mainly introduce the applications of genome editing in constructing animal disease models and gene therapies, as well as its future prospects and challenges. Keywords: Genome editing, Disease models, Gene therapy, CRISPR-Cas9 |
| first_indexed | 2024-12-21T02:42:06Z |
| format | Article |
| id | doaj.art-7ad4ae5e51ff403085b9ac6727974107 |
| institution | Directory Open Access Journal |
| issn | 2001-0370 |
| language | English |
| last_indexed | 2024-12-21T02:42:06Z |
| publishDate | 2019-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Computational and Structural Biotechnology Journal |
| spelling | doaj.art-7ad4ae5e51ff403085b9ac67279741072022-12-21T19:18:40ZengElsevierComputational and Structural Biotechnology Journal2001-03702019-01-0117689698Applications of Genome Editing Technology in Animal Disease Modeling and Gene TherapyQian Li0Zhou Qin1Qingnan Wang2Ting Xu3Yang Yang4Zhiyao He5State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, ChinaState Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, China; Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, ChinaState Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, ChinaState Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, China; Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, ChinaState Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, China; Corresponding authors at: State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, No. 17, Section 3, Renmin Road South, Chengdu, Sichuan 610041, China.State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, China; Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center, Chengdu, Sichuan 610041, China; Corresponding authors at: State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, No. 17, Section 3, Renmin Road South, Chengdu, Sichuan 610041, China.Genome editing technology is a technique for targeted genetic modifications, enabling the knockout and addition of specific DNA fragments. This technology has been widely used in various types of biomedical research, clinics and agriculture. In terms of disease research, constructing appropriate animal models is necessary. Combining reproductive technology with genome editing, many animal disease models have been generated for basic and clinical research. In addition, precisely targeted modifications allow genome editing to flourish in the field of gene therapy. Many mutations refractory to traditional gene therapy could be permanently corrected at the DNA level. Thus, genome editing is undoubtedly a promising technology for gene therapy. In this review, we mainly introduce the applications of genome editing in constructing animal disease models and gene therapies, as well as its future prospects and challenges. Keywords: Genome editing, Disease models, Gene therapy, CRISPR-Cas9http://www.sciencedirect.com/science/article/pii/S2001037019300650 |
| spellingShingle | Qian Li Zhou Qin Qingnan Wang Ting Xu Yang Yang Zhiyao He Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy Computational and Structural Biotechnology Journal |
| title | Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy |
| title_full | Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy |
| title_fullStr | Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy |
| title_full_unstemmed | Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy |
| title_short | Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy |
| title_sort | applications of genome editing technology in animal disease modeling and gene therapy |
| url | http://www.sciencedirect.com/science/article/pii/S2001037019300650 |
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