Lipid nanoparticle technology for therapeutic gene regulation in the liver

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dc.creatorWitzigmann, Dominik
dc.creatorKulkarni, Jayesh A.
dc.creatorLeung, Jerry
dc.creatorChen, Sam
dc.creatorCullis, Pieter R.
dc.creatorvan der Meel, Roy
dc.date.accessioned2020-07-21T18:15:06Z
dc.date.available2020-07-21T18:15:06Z
dc.date.created2020-06-25
dc.description.abstractenglishHereditary genetic disorders, cancer, and infectious diseases of the liver affect millions of people around the globe and are a major public health burden. Most contemporary treatments offer limited relief as they generally aim to alleviate disease symptoms. Targeting the root cause of diseases originating in the liver by regulating malfunctioning genes with nucleic acid-based drugs holds great promise as a therapeutic approach. However, employing nucleic acid therapeutics in vivo is challenging due to their unfavorable characteristics. Lipid nanoparticle (LNP) delivery technology is a revolutionary development that has enabled clinical translation of gene therapies. LNPs can deliver siRNA, mRNA, DNA, or gene-editing complexes, providing opportunities to treat hepatic diseases by silencing pathogenic genes, expressing therapeutic proteins, or correcting genetic defects. Here we discuss the state-of-the-art LNP technology for hepatic gene therapy including formulation design parameters, production methods, preclinical development and clinical translation.spa
dc.format.extent49 páginasspa
dc.format.mimetypeapplication/pdfspa
dc.identifier.doihttps://doi.org/10.1016/j.addr.2020.06.026spa
dc.identifier.issn0169-409Xspa
dc.identifier.otherhttps://www.sciencedirect.com/science/article/pii/S0169409X20300727?via%3Dihub#ks0005spa
dc.identifier.urihttps://hdl.handle.net/20.500.12010/10876
dc.publisherAdvanced Drug Delivery Reviewseng
dc.rights.accessrightsinfo:eu-repo/semantics/openAccessspa
dc.sourcereponame:Expeditio Repositorio Institucional UJTLspa
dc.sourceinstname:Universidad de Bogotá Jorge Tadeo Lozanospa
dc.subjectGene therapyspa
dc.subjectliverspa
dc.subjectlipid nanoparticle (LNP)spa
dc.subjectlipidsspa
dc.subjecthepatocytespa
dc.subjectsmall interfering RNA (siRNA)spa
dc.subjectmessenger RNA (mRNA)spa
dc.subjectDNAspa
dc.subjectguide RNA (gRNA)spa
dc.subjectCRISPR/Cas9spa
dc.subjectgene silencingspa
dc.subjectgene expressionspa
dc.subjectgene editingspa
dc.subject.lembSíndrome respiratorio agudo gravespa
dc.subject.lembCOVID-19spa
dc.subject.lembSARS-CoV-2spa
dc.subject.lembCoronavirusspa
dc.titleLipid nanoparticle technology for therapeutic gene regulation in the liverspa
dc.type.hasversioninfo:eu-repo/semantics/acceptedVersionspa
dc.type.localArtículospa

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