Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives
Abstract The expanding genome editing toolbox has revolutionized life science research ranging from the bench to the bedside. These “molecular scissors” have offered us unprecedented abilities to manipulate nucleic acid sequences precisely in living cells from diverse species. Continued advances in...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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Wiley
2022-09-01
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| Series: | MedComm |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/mco2.155 |
| _version_ | 1798004228469293056 |
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| author | Weilin Zhou Jinrong Yang Yalan Zhang Xiaoyi Hu Wei Wang |
| author_facet | Weilin Zhou Jinrong Yang Yalan Zhang Xiaoyi Hu Wei Wang |
| author_sort | Weilin Zhou |
| collection | DOAJ |
| description | Abstract The expanding genome editing toolbox has revolutionized life science research ranging from the bench to the bedside. These “molecular scissors” have offered us unprecedented abilities to manipulate nucleic acid sequences precisely in living cells from diverse species. Continued advances in genome editing exponentially broaden our knowledge of human genetics, epigenetics, molecular biology, and pathology. Currently, gene editing‐mediated therapies have led to impressive responses in patients with hematological diseases, including sickle cell disease and thalassemia. With the discovery of more efficient, precise and sophisticated gene‐editing tools, more therapeutic gene‐editing approaches will enter the clinic to treat various diseases, such as acquired immunodeficiency sydrome (AIDS), hematologic malignancies, and even severe acute respiratory syndrome coronavirus 2 (SARS‐CoV‐2) infection. These initial successes have spurred the further innovation and development of gene‐editing technology. In this review, we will introduce the architecture and mechanism of the current gene‐editing tools, including clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR‐associated nuclease‐based tools and other protein‐based DNA targeting systems, and we summarize the meaningful applications of diverse technologies in preclinical studies, focusing on the establishment of disease models and diagnostic techniques. Finally, we provide a comprehensive overview of clinical information using gene‐editing therapeutics for treating various human diseases and emphasize the opportunities and challenges. |
| first_indexed | 2024-04-11T12:20:24Z |
| format | Article |
| id | doaj.art-6197d812ed074c3d80519ef4cba3ca8e |
| institution | Directory Open Access Journal |
| issn | 2688-2663 |
| language | English |
| last_indexed | 2024-04-11T12:20:24Z |
| publishDate | 2022-09-01 |
| publisher | Wiley |
| record_format | Article |
| series | MedComm |
| spelling | doaj.art-6197d812ed074c3d80519ef4cba3ca8e2022-12-22T04:24:06ZengWileyMedComm2688-26632022-09-0133n/an/a10.1002/mco2.155Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectivesWeilin Zhou0Jinrong Yang1Yalan Zhang2Xiaoyi Hu3Wei Wang4Department of Biotherapyy State Key Laboratory of Biotherapy and Cancer Center West China Hospital Sichuan University Chengdu People's Republic of ChinaDepartment of Biotherapyy State Key Laboratory of Biotherapy and Cancer Center West China Hospital Sichuan University Chengdu People's Republic of ChinaDepartment of Biotherapyy State Key Laboratory of Biotherapy and Cancer Center West China Hospital Sichuan University Chengdu People's Republic of ChinaDepartment of Biotherapyy State Key Laboratory of Biotherapy and Cancer Center West China Hospital Sichuan University Chengdu People's Republic of ChinaDepartment of Biotherapyy State Key Laboratory of Biotherapy and Cancer Center West China Hospital Sichuan University Chengdu People's Republic of ChinaAbstract The expanding genome editing toolbox has revolutionized life science research ranging from the bench to the bedside. These “molecular scissors” have offered us unprecedented abilities to manipulate nucleic acid sequences precisely in living cells from diverse species. Continued advances in genome editing exponentially broaden our knowledge of human genetics, epigenetics, molecular biology, and pathology. Currently, gene editing‐mediated therapies have led to impressive responses in patients with hematological diseases, including sickle cell disease and thalassemia. With the discovery of more efficient, precise and sophisticated gene‐editing tools, more therapeutic gene‐editing approaches will enter the clinic to treat various diseases, such as acquired immunodeficiency sydrome (AIDS), hematologic malignancies, and even severe acute respiratory syndrome coronavirus 2 (SARS‐CoV‐2) infection. These initial successes have spurred the further innovation and development of gene‐editing technology. In this review, we will introduce the architecture and mechanism of the current gene‐editing tools, including clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR‐associated nuclease‐based tools and other protein‐based DNA targeting systems, and we summarize the meaningful applications of diverse technologies in preclinical studies, focusing on the establishment of disease models and diagnostic techniques. Finally, we provide a comprehensive overview of clinical information using gene‐editing therapeutics for treating various human diseases and emphasize the opportunities and challenges.https://doi.org/10.1002/mco2.155clinical trialsdisease diagnosisdisease modelinggene therapygenome editingimmunotherapy |
| spellingShingle | Weilin Zhou Jinrong Yang Yalan Zhang Xiaoyi Hu Wei Wang Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives MedComm clinical trials disease diagnosis disease modeling gene therapy genome editing immunotherapy |
| title | Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives |
| title_full | Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives |
| title_fullStr | Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives |
| title_full_unstemmed | Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives |
| title_short | Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives |
| title_sort | current landscape of gene editing technology in biomedicine applications advantages challenges and perspectives |
| topic | clinical trials disease diagnosis disease modeling gene therapy genome editing immunotherapy |
| url | https://doi.org/10.1002/mco2.155 |
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