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dc.creatorYang, Tongren
dc.creatorLi, Chunhui
dc.creatorWang, Xiaoxia
dc.creatorZhao, Deyao
dc.creatorZhang, Mengjie
dc.creatorCao, Huiqing
dc.creatorLiang, Zicai
dc.creatorXiao, Haihua
dc.creatorLiang, Xing-Jie
dc.creatorWeng, Yuhua
dc.creatorHuang, Yuanyu
dc.date.accessioned2020-07-23T15:21:11Z
dc.date.available2020-07-23T15:21:11Z
dc.date.created2020
dc.identifier.issn2452-199Xspa
dc.identifier.otherhttps://doi.org/10.1016/j.bioactmat.2020.07.003spa
dc.identifier.urihttp://hdl.handle.net/20.500.12010/11028
dc.description.abstractmRNA is a novel class of therapeutic modality that holds great promise in vaccination, protein replacement therapy, cancer immunotherapy, immune cell engineering etc. However, optimization of mRNA molecules and efficient in vivo delivery are quite important but challenging for its broad application. Here we present an ionizable lipid nanoparticle (iLNP) based on iBL0713 lipid for in vitro and in vivo expression of desired proteins using codon-optimized mRNAs. mRNAs encoding luciferase or erythropoietin (EPO) were prepared by in vitro transcription and formulated with proposed iLNP, to form iLP171/mRNA formulations. It was revealed that both luciferase and EPO proteins were successfully expressed by human hepatocellular carcinoma cells and hepatocytes. The maximum amount of protein expression was found at 6 h post-administration. The expression efficiency of EPO with codon-optimized mRNA was significantly higher than that of unoptimized mRNA. Moreover, no toxicity or immunogenicity was observed for these mRNA formulations. Therefore, our study provides a useful and promising platform for mRNA therapeutic development.spa
dc.format.extent9 páginasspa
dc.format.mimetypeapplication/pdfspa
dc.publisherScience Directeng
dc.sourcereponame:Expeditio Repositorio Institucional UJTLspa
dc.sourceinstname:Universidad de Bogotá Jorge Tadeo Lozanospa
dc.subjectmRNA therapyspa
dc.subjectLipid nanoparticlespa
dc.subjectmRNA deliveryspa
dc.subjectErythropoietinspa
dc.subjectCodon optimizationspa
dc.titleEfficient hepatic delivery and protein expression enabled by optimized mRNA and ionizable lipid nanoparticlespa
dc.type.localArtículospa
dc.subject.lembSíndrome respiratorio agudo gravespa
dc.subject.lembCOVID-19spa
dc.subject.lembSARS-CoV-2spa
dc.subject.lembCoronavirusspa
dc.rights.accessrightsinfo:eu-repo/semantics/openAccessspa
dc.type.hasversioninfo:eu-repo/semantics/acceptedVersionspa
dc.identifier.doihttps://doi.org/10.1016/j.bioactmat.2020.07.003spa


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