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Adenine Base Editor Ribonucleoproteins Delivered by Lentivirus-Like Particles Show High On-Target Base Editing and Undetectable RNA Off-Target Activities

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dc.contributor.authorLyu, Pin-
dc.contributor.authorLu, Zuyan-
dc.contributor.authorSung-Ik Cho-
dc.contributor.authorYadav, Manish-
dc.contributor.authorYoo, Kyung Whan-
dc.contributor.authorAtala, Anthony-
dc.contributor.authorJin-Soo Kim-
dc.contributor.authorLu, Baisong-
dc.date.accessioned2021-07-05T01:30:08Z-
dc.date.accessioned2021-07-05T01:30:08Z-
dc.date.available2021-07-05T01:30:08Z-
dc.date.available2021-07-05T01:30:08Z-
dc.date.created2021-04-21-
dc.date.issued2021-02-
dc.identifier.issn2573-1599-
dc.identifier.urihttps://pr.ibs.re.kr/handle/8788114/9835-
dc.description.abstractAdenine base editors (ABEs) can correct gene mutations without creating double-strand breaks. However, in recent reports, these editors showed guide-independent RNA off-target activities. This work describes our development of a delivery method to minimize ABEs' RNA off-target activity. After discovering a RNA off-target hot spot for sensitive detection of RNA off-target activities, we found that delivering ribonucleoproteins (RNPs) by electroporation generated undetectable non-specific RNA editing, but on-target base editing activity was also relatively low. We then explored a lentivirus capsid-based delivery strategy to deliver ABE. We used aptamer/aptamer-binding protein (ABP) interactions to package ABE RNPs into lentiviral capsids. Capsid RNPs were delivered to human cells for highly efficient guided base editing. Importantly, RNA off-target activities from the capsid RNPs were undetectable. Our new lentiviral capsid-based ABE RNP delivery method with minimal RNA off-target activities makes ABE one step closer to possible therapeutic applications.-
dc.language영어-
dc.publisherMARY ANN LIEBERT, INC-
dc.titleAdenine Base Editor Ribonucleoproteins Delivered by Lentivirus-Like Particles Show High On-Target Base Editing and Undetectable RNA Off-Target Activities-
dc.typeArticle-
dc.type.rimsART-
dc.identifier.wosid000621616400012-
dc.identifier.scopusid2-s2.0-85098766024-
dc.identifier.rimsid75363-
dc.contributor.affiliatedAuthorSung-Ik Cho-
dc.contributor.affiliatedAuthorJin-Soo Kim-
dc.identifier.doi10.1089/crispr.2020.0095-
dc.identifier.bibliographicCitationCRISPR JOURNAL, v.4, no.1, pp.69 - 81-
dc.relation.isPartOfCRISPR JOURNAL-
dc.citation.titleCRISPR JOURNAL-
dc.citation.volume4-
dc.citation.number1-
dc.citation.startPage69-
dc.citation.endPage81-
dc.type.docTypeArticle-
dc.description.journalClass1-
dc.description.journalClass1-
dc.description.isOpenAccessN-
dc.description.journalRegisteredClassscie-
dc.description.journalRegisteredClassscopus-
dc.relation.journalResearchAreaGenetics & Heredity-
dc.relation.journalWebOfScienceCategoryGenetics & Heredity-
Appears in Collections:
Center for Genome Engineering(유전체 교정 연구단) > 1. Journal Papers (저널논문)
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