Full metadata record
DC Field | Value | Language |
---|---|---|
dc.contributor.author | 장준 | * |
dc.contributor.author | 이혁진 | * |
dc.date.accessioned | 2022-02-22T16:30:55Z | - |
dc.date.available | 2022-02-22T16:30:55Z | - |
dc.date.issued | 2022 | * |
dc.identifier.issn | 2375-2548 | * |
dc.identifier.other | OAK-30888 | * |
dc.identifier.uri | https://dspace.ewha.ac.kr/handle/2015.oak/260550 | - |
dc.description.abstract | Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic effects and treatment options for inhibitor patients. Antithrombin (AT), an endogenous negative regulator of thrombin generation, is a potent genome editing target for sustainable treatment of patients with hemophilia A and B. In this study, we developed and optimized lipid nanoparticles (LNPs) to deliver Cas9 mRNA along with single guide RNA that targeted AT in the mouse liver. The LNP-mediated CRISPR-Cas9 delivery resulted in the inhibition of AT that led to improvement in thrombin generation. Bleeding-associated phenotypes were recovered in both hemophilia A and B mice. No active off-targets, liver-induced toxicity, and substantial anti-Cas9 immune responses were detected, indicating that the LNP-mediated CRISPR-Cas9 delivery was a safe and efficient approach for hemophilia therapy. Copyright © 2022 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works. Distributed under a Creative Commons Attribution NonCommercial License 4.0 (CC BY-NC). | * |
dc.language | English | * |
dc.publisher | American Association for the Advancement of Science | * |
dc.title | In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy | * |
dc.type | Article | * |
dc.relation.issue | 3 | * |
dc.relation.volume | 8 | * |
dc.relation.index | SCIE | * |
dc.relation.index | SCOPUS | * |
dc.relation.journaltitle | Science Advances | * |
dc.identifier.doi | 10.1126/sciadv.abj6901 | * |
dc.identifier.wosid | WOS:000745886100014 | * |
dc.identifier.scopusid | 2-s2.0-85123284733 | * |
dc.author.google | Han J.P. | * |
dc.author.google | Kim M. | * |
dc.author.google | Choi B.S. | * |
dc.author.google | Lee J.H. | * |
dc.author.google | Lee G.S. | * |
dc.author.google | Jeong M. | * |
dc.author.google | Lee Y. | * |
dc.author.google | Kim E.-A. | * |
dc.author.google | Oh H.-K. | * |
dc.author.google | Go N. | * |
dc.author.google | Lee H. | * |
dc.author.google | Lee K.J. | * |
dc.author.google | Kim U.G. | * |
dc.author.google | Lee J.Y. | * |
dc.author.google | Kim S. | * |
dc.author.google | Chang J. | * |
dc.author.google | Song D.W. | * |
dc.author.google | Yeom S.C. | * |
dc.contributor.scopusid | 장준(8735999100) | * |
dc.contributor.scopusid | 이혁진(55233457200) | * |
dc.date.modifydate | 20240220111730 | * |