Gene editing in monogenic autism spectrum disorder: animal models and gene therapies
Autism spectrum disorder (ASD) is a lifelong neurodevelopmental disease, and its diagnosis is dependent on behavioral manifestation, such as impaired reciprocal social interactions, stereotyped repetitive behaviors, as well as restricted interests. However, ASD etiology has eluded researchers to dat...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2022-12-01
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| Series: | Frontiers in Molecular Neuroscience |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fnmol.2022.1043018/full |
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| author | Na Wang Longbao Lv Xiaoyi Huang Mingqin Shi Youwu Dai Yuanyuan Wei Bonan Xu Chenyang Fu Haoyu Huang Hongling Shi Yun Liu Xintian Hu Dongdong Qin |
| author_facet | Na Wang Longbao Lv Xiaoyi Huang Mingqin Shi Youwu Dai Yuanyuan Wei Bonan Xu Chenyang Fu Haoyu Huang Hongling Shi Yun Liu Xintian Hu Dongdong Qin |
| author_sort | Na Wang |
| collection | DOAJ |
| description | Autism spectrum disorder (ASD) is a lifelong neurodevelopmental disease, and its diagnosis is dependent on behavioral manifestation, such as impaired reciprocal social interactions, stereotyped repetitive behaviors, as well as restricted interests. However, ASD etiology has eluded researchers to date. In the past decades, based on strong genetic evidence including mutations in a single gene, gene editing technology has become an essential tool for exploring the pathogenetic mechanisms of ASD via constructing genetically modified animal models which validates the casual relationship between genetic risk factors and the development of ASD, thus contributing to developing ideal candidates for gene therapies. The present review discusses the progress in gene editing techniques and genetic research, animal models established by gene editing, as well as gene therapies in ASD. Future research should focus on improving the validity of animal models, and reliable DNA diagnostics and accurate prediction of the functional effects of the mutation will likely be equally crucial for the safe application of gene therapies. |
| first_indexed | 2024-04-12T01:43:45Z |
| format | Article |
| id | doaj.art-367b1401de3949d4a9cffb80a91efcbe |
| institution | Directory Open Access Journal |
| issn | 1662-5099 |
| language | English |
| last_indexed | 2024-04-12T01:43:45Z |
| publishDate | 2022-12-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Molecular Neuroscience |
| spelling | doaj.art-367b1401de3949d4a9cffb80a91efcbe2022-12-22T03:53:08ZengFrontiers Media S.A.Frontiers in Molecular Neuroscience1662-50992022-12-011510.3389/fnmol.2022.10430181043018Gene editing in monogenic autism spectrum disorder: animal models and gene therapiesNa Wang0Longbao Lv1Xiaoyi Huang2Mingqin Shi3Youwu Dai4Yuanyuan Wei5Bonan Xu6Chenyang Fu7Haoyu Huang8Hongling Shi9Yun Liu10Xintian Hu11Dongdong Qin12School of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaKey Laboratory of Animal Models and Human Disease Mechanisms of the Chinese Academy of Sciences and Yunnan Province, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming, Yunnan, ChinaSchool of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaSchool of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaSchool of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaSchool of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaSchool of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaSchool of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaDepartment of Pediatric Rehabilitation Medicine, Kunming Children’s Hospital, Kunming, Yunnan, ChinaDepartment of Rehabilitation Medicine, The Third People’s Hospital of Yunnan Province, Kunming, Yunnan, ChinaDepartment of Pediatric Rehabilitation Medicine, Kunming Children’s Hospital, Kunming, Yunnan, ChinaKey Laboratory of Animal Models and Human Disease Mechanisms of the Chinese Academy of Sciences and Yunnan Province, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming, Yunnan, ChinaSchool of Basic Medical Sciences, Yunnan University of Chinese Medicine, Kunming, Yunnan, ChinaAutism spectrum disorder (ASD) is a lifelong neurodevelopmental disease, and its diagnosis is dependent on behavioral manifestation, such as impaired reciprocal social interactions, stereotyped repetitive behaviors, as well as restricted interests. However, ASD etiology has eluded researchers to date. In the past decades, based on strong genetic evidence including mutations in a single gene, gene editing technology has become an essential tool for exploring the pathogenetic mechanisms of ASD via constructing genetically modified animal models which validates the casual relationship between genetic risk factors and the development of ASD, thus contributing to developing ideal candidates for gene therapies. The present review discusses the progress in gene editing techniques and genetic research, animal models established by gene editing, as well as gene therapies in ASD. Future research should focus on improving the validity of animal models, and reliable DNA diagnostics and accurate prediction of the functional effects of the mutation will likely be equally crucial for the safe application of gene therapies.https://www.frontiersin.org/articles/10.3389/fnmol.2022.1043018/fullautism spectrum disordergene editinganimal modelpathogenesistherapy |
| spellingShingle | Na Wang Longbao Lv Xiaoyi Huang Mingqin Shi Youwu Dai Yuanyuan Wei Bonan Xu Chenyang Fu Haoyu Huang Hongling Shi Yun Liu Xintian Hu Dongdong Qin Gene editing in monogenic autism spectrum disorder: animal models and gene therapies Frontiers in Molecular Neuroscience autism spectrum disorder gene editing animal model pathogenesis therapy |
| title | Gene editing in monogenic autism spectrum disorder: animal models and gene therapies |
| title_full | Gene editing in monogenic autism spectrum disorder: animal models and gene therapies |
| title_fullStr | Gene editing in monogenic autism spectrum disorder: animal models and gene therapies |
| title_full_unstemmed | Gene editing in monogenic autism spectrum disorder: animal models and gene therapies |
| title_short | Gene editing in monogenic autism spectrum disorder: animal models and gene therapies |
| title_sort | gene editing in monogenic autism spectrum disorder animal models and gene therapies |
| topic | autism spectrum disorder gene editing animal model pathogenesis therapy |
| url | https://www.frontiersin.org/articles/10.3389/fnmol.2022.1043018/full |
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