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dc.contributor.author유지현-
dc.date.accessioned2019-10-21T02:24:06Z-
dc.date.available2019-10-21T02:24:06Z-
dc.date.issued2019-08-
dc.identifier.citationMOLECULAR THERAPY, v. 27, NO 8, Page. 1364-1371en_US
dc.identifier.issn1525-0016-
dc.identifier.issn1525-0024-
dc.identifier.urihttps://www.sciencedirect.com/science/article/pii/S1525001619302278?via%3Dihub-
dc.identifier.urihttps://repository.hanyang.ac.kr/handle/20.500.11754/111251-
dc.description.abstractA nonsense mutation is a substitutive mutation in a DNA sequence that causes a premature termination during translation and produces stalled proteins, resulting in dysfunction of a gene. Although it usually induces severe genetic disorders, there are no definite methods for inducing read through of premature termination codons (PTCs). Here, we present a targeted tool for bypassing PTCs, named CRISPR-pass, that uses CRISPR-mediated adenine base editors. CRISPR-pass, which should be applicable to 95.5% of clinically significant nonsense mutations in the ClinVar database, rescues protein synthesis in patient-derived fibroblasts, suggesting potential clinical utility.en_US
dc.description.sponsorshipThis work was supported by National Research Foundation of Korea (NRF) grants (2017R1A6A3A04004741 to D.H.J.; 2015M3A9E 6028949, 2017M3A9B4062654, and 2018M3D1A1058826 to Jeong H. Kim; and 2018M3A9H3022412 to S. B.); a grant from the Korea Research Institute of Standards and Science (KRISS - 2018 - GP2018-0018 to Jeong H. Kim); and grants from the Next Generation BioGreen 21 Program (PJ01319301), Korea Healthcare Technology R&D Project (HI16C1012), and Technology Innovation Program (20000158) (to S.B.).en_US
dc.language.isoenen_US
dc.publisherCELL PRESSen_US
dc.subjectGENOMIC DNAen_US
dc.subjectCELLSen_US
dc.titleCRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editorsen_US
dc.typeArticleen_US
dc.relation.no8-
dc.relation.volume27-
dc.identifier.doi10.1016/j.ymthe.2019.05.013-
dc.relation.page1016-1016-
dc.relation.journalMOLECULAR THERAPY-
dc.contributor.googleauthorLee, Choongil-
dc.contributor.googleauthorJo, Dong Hyun-
dc.contributor.googleauthorHwang, Gue-Ho-
dc.contributor.googleauthorYu, Jihyeon-
dc.contributor.googleauthorKim, Jin Hyoung-
dc.contributor.googleauthorPark, Se-eun-
dc.contributor.googleauthorKim, Jin-Soo-
dc.contributor.googleauthorKim, Jeong Hun-
dc.contributor.googleauthorBae, Sangsu-
dc.relation.code2019002340-
dc.sector.campusS-
dc.sector.daehakRESEARCH INSTITUTE[S]-
dc.sector.departmentRESEARCH INSTITUTE FOR CONVERGENCE OF BASIC SCIENCES-
dc.identifier.pidmuner00-
dc.identifier.researcherIDU-4150-2019-
dc.identifier.orcidhttp://orcid.org/0000-0003-0247-7753-
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