{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T12:28:08Z","timestamp":1793363288687,"version":"4.5.0"},"reference-count":54,"publisher":"SAGE Publications","issue":"1","license":[{"start":{"date-parts":[[2022,2,1]],"date-time":"2022-02-01T00:00:00Z","timestamp":1643673600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["The CRISPR Journal"],"published-print":{"date-parts":[[2022,2,1]]},"abstract":"<jats:p>\n                    Mutations in specific genes, including synuclein alpha (\n                    <jats:italic toggle=\"yes\">SNCA<\/jats:italic>\n                    ) that encodes the \u03b1-synuclein protein, are known to be risk factors for sporadic Parkinson's disease (PD), as well as critical factors for familial PD. In particular, A53T-mutated\n                    <jats:italic toggle=\"yes\">SNCA<\/jats:italic>\n                    (A53T-SNCA) is a well-studied familial pathologic mutation in PD. However, techniques for deletion of the mutated\n                    <jats:italic toggle=\"yes\">SNCA<\/jats:italic>\n                    gene\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    have not been developed. Here, we used the CRISPR-Cas9 system to delete A53T-SNCA\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    as well as\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    . Adeno-associated virus carrying SaCas9-KKH with a single-guide RNA targeting A53T-SNCA significantly reduced A53T-SNCA expression levels\n                    <jats:italic toggle=\"yes\">in vitro<\/jats:italic>\n                    . Furthermore, we tested its therapeutic potential\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    in a viral A53T-SNCA-overexpressing rat model of PD. Gene deletion of A53T-SNCA significantly rescued the overexpression of \u03b1-synuclein, reactive microgliosis, dopaminergic neurodegeneration, and parkinsonian motor symptoms. Our findings propose CRISPR-Cas9 system as a potential prevention strategy for A53T-SNCA-specific PD.\n                  <\/jats:p>","DOI":"10.1089\/crispr.2021.0025","type":"journal-article","created":{"date-parts":[[2022,2,22]],"date-time":"2022-02-22T10:08:59Z","timestamp":1645524539000},"page":"95-108","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":64,"title":["CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease\n                    <i>In Vivo<\/i>"],"prefix":"10.1177","volume":"5","author":[{"given":"Hyung Ho","family":"Yoon","sequence":"first","affiliation":[{"name":"Department of Neurological Surgery, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sunghyeok","family":"Ye","sequence":"additional","affiliation":[{"name":"RnD center, GeneCker, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sunhwa","family":"Lim","sequence":"additional","affiliation":[{"name":"Convergence Research Center for Dementia, Korea Institute of Science and Technology, Seoul, Korea; Kyung Hee University, Seoul, Korea."},{"name":"Brain Science Institute, Korea Institute of Science and Technology, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ara","family":"Jo","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery, College of Medicine, Korea University, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hawon","family":"Lee","sequence":"additional","affiliation":[{"name":"RnD center, GeneCker, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Felix","family":"Hong","sequence":"additional","affiliation":[{"name":"RnD center, GeneCker, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seung Eun","family":"Lee","sequence":"additional","affiliation":[{"name":"Virus Facility, Research Animal Resource Center, Korea Institute of Science and Technology, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Soo-Jin","family":"Oh","sequence":"additional","affiliation":[{"name":"Convergence Research Center for Dementia, Korea Institute of Science and Technology, Seoul, Korea; Kyung Hee University, Seoul, Korea."},{"name":"Brain Science Institute, Korea Institute of Science and Technology, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Na-Rae","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences and Department of Physiology, College of Medicine, Korea University, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kyoungmi","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences and Department of Physiology, College of Medicine, Korea University, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bum-Joon","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery, College of Medicine, Korea University, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hyunjin","family":"Kim","sequence":"additional","affiliation":[{"name":"Brain Science Institute, Korea Institute of Science and Technology, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. Justin","family":"Lee","sequence":"additional","affiliation":[{"name":"Center for Cognition and Sociality, Institute for Basic Science, Daejeon, Korea; and Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Min-Ho","family":"Nam","sequence":"additional","affiliation":[{"name":"Brain Science Institute, Korea Institute of Science and Technology, Seoul, Korea; Kyung Hee University, Seoul, Korea."},{"name":"Department of KHU-KIST Convergence Science and Technology, Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Junseok W.","family":"Hur","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery, College of Medicine, Korea University, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sang Ryong","family":"Jeon","sequence":"additional","affiliation":[{"name":"Department of Neurological Surgery, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea; Kyung Hee University, Seoul, Korea."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2022,2,22]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0896-6273(03)00568-3"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1038\/ng.485"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1038\/ng.487"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318264e353"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.276.5321.2045"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.67"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jneumeth.2015.07.025"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1186\/s40478-017-0416-x"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.3967-11.2011"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.cub.2019.11.079"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.22-07-02780.2002"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1038\/nbt.2507"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1038\/nbt.2508"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.5483\/BMBRep.2019.52.8.149"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1007\/s13258-019-00830-w"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrg2988"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrg2971"},{"key":"e_1_3_5_19_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrmicro1266"},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1002\/biot.201400046"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.1038\/nature14299"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.aad5143"},{"key":"e_1_3_5_23_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.aad5725"},{"key":"e_1_3_5_24_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.aad5177"},{"key":"e_1_3_5_25_2","doi-asserted-by":"publisher","DOI":"10.1038\/nbt.3404"},{"key":"e_1_3_5_26_2","doi-asserted-by":"publisher","DOI":"10.5483\/BMBRep.2019.52.8.149"},{"key":"e_1_3_5_27_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-018-0209-1"},{"key":"e_1_3_5_28_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.tibtech.2019.03.008"},{"key":"e_1_3_5_29_2","doi-asserted-by":"publisher","DOI":"10.1038\/nbt.3471"},{"key":"e_1_3_5_30_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2031054"},{"key":"e_1_3_5_31_2","doi-asserted-by":"publisher","DOI":"10.15283\/ijsc18110"},{"key":"e_1_3_5_32_2","doi-asserted-by":"publisher","DOI":"10.1007\/s10571-019-00761-w"},{"key":"e_1_3_5_33_2","doi-asserted-by":"publisher","DOI":"10.1007\/s11481-019-09844-3"},{"key":"e_1_3_5_34_2","doi-asserted-by":"publisher","DOI":"10.1038\/ncomms12932"},{"key":"e_1_3_5_35_2","doi-asserted-by":"publisher","DOI":"10.1038\/nature14592"},{"key":"e_1_3_5_36_2","first-page":"10272","article-title":"Mb-and FnCpf1 nucleases are active in mammalian cells: activities and PAM preferences of four wild-type Cpf1 nucleases and of their altered PAM specificity variants","volume":"46","author":"T\u00f3th E","year":"2018","unstructured":"T\u00f3th E, , Czene BC, , Kulcs\u00e1r PI, et al. Mb-and FnCpf1 nucleases are active in mammalian cells: activities and PAM preferences of four wild-type Cpf1 nucleases and of their altered PAM specificity variants. Nucleic Acids Res, 2018; 46:10272\u201310285. 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