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Enhancement of Gene Editing and Base Editing with Therapeutic Ribonucleoproteins through In Vivo Delivery Based on Absorptive Silica Nanoconstruct

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dc.contributor.authorSeongchan Kim-
dc.contributor.authorYou Kyeong Jeong-
dc.contributor.authorChang Sik Cho-
dc.contributor.authorSeokHoon Lee-
dc.contributor.authorChang Ho Sohn-
dc.contributor.authorJeong Hun Kim-
dc.contributor.authorYoungdo Jeong-
dc.contributor.authorDong Hyun Jo-
dc.contributor.authorSangsu Bae-
dc.contributor.authorHyojin Lee-
dc.date.accessioned2023-02-13T22:00:57Z-
dc.date.available2023-02-13T22:00:57Z-
dc.date.created2022-11-29-
dc.date.issued2023-02-
dc.identifier.issn2192-2640-
dc.identifier.urihttps://pr.ibs.re.kr/handle/8788114/13015-
dc.description.abstractKey to the widespread and secure application of genome editing tools is the safe and effective delivery of multiple components of ribonucleoproteins (RNPs) into single cells, which remains a biological barrier to their clinical application. To overcome this issue, a robust RNP delivery platform based on a biocompatible sponge-like silica nanoconstruct (SN) for storing and directly delivering therapeutic RNPs, including Cas9 nuclease RNP (Cas9-RNP) and base editor RNP (BE-RNP) is designed. Compared with commercialized material such as lipid-based methods, up to 50-fold gene deletion and 10-fold base substitution efficiency is obtained with a low off-target efficiency by targeting various cells and genes. In particular, gene correction is successfully induced by SN-based delivery through intravenous injection in an in vivo solid-tumor model and through subretinal injection in mouse eye. Moreover, because of its low toxicity and high biodegradability, SN has negligible effect on cellular function of organs. As the engineered SN can overcome practical challenges associated with therapeutic RNP application, it is strongly expected this platform to be a modular RNPs delivery system, facilitating in vivo gene deletion and editing.-
dc.language영어-
dc.publisherWILEY-
dc.titleEnhancement of Gene Editing and Base Editing with Therapeutic Ribonucleoproteins through In Vivo Delivery Based on Absorptive Silica Nanoconstruct-
dc.typeArticle-
dc.type.rimsART-
dc.identifier.wosid000880711900001-
dc.identifier.scopusid2-s2.0-85141973993-
dc.identifier.rimsid79275-
dc.contributor.affiliatedAuthorChang Ho Sohn-
dc.identifier.doi10.1002/adhm.202201825-
dc.identifier.bibliographicCitationADVANCED HEALTHCARE MATERIALS, v.12, no.4-
dc.relation.isPartOfADVANCED HEALTHCARE MATERIALS-
dc.citation.titleADVANCED HEALTHCARE MATERIALS-
dc.citation.volume12-
dc.citation.number4-
dc.description.journalClass1-
dc.description.journalClass1-
dc.description.isOpenAccessN-
dc.description.journalRegisteredClassscie-
dc.description.journalRegisteredClassscopus-
dc.relation.journalWebOfScienceCategoryEngineering, Biomedical-
dc.relation.journalWebOfScienceCategoryNanoscience & Nanotechnology-
dc.relation.journalWebOfScienceCategoryMaterials Science, Biomaterials-
dc.subject.keywordPlusCAS9 RIBONUCLEOPROTEIN-
dc.subject.keywordPlusGENOMIC DNA-
dc.subject.keywordPlusCRISPR/CAS9-
dc.subject.keywordPlusTARGET-
dc.subject.keywordPlusSTRATEGIES-
dc.subject.keywordPlusDESIGN-
dc.subject.keywordPlusCELLS-
dc.subject.keywordPlusTOOL-
dc.subject.keywordAuthorbase editing-
dc.subject.keywordAuthorgene editing-
dc.subject.keywordAuthorporous nanoparticles-
dc.subject.keywordAuthorribonucleoproteins delivery-
dc.subject.keywordAuthortargeted tumor therapy-
Appears in Collections:
Center for Nanomedicine (나노의학 연구단) > 1. Journal Papers (저널논문)
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