Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8842166 | Progress in Neurobiology | 2018 | 17 Pages |
Abstract
Accurate and timely expression of specific genes guarantees the healthy development and function of the brain. Indeed, variations in the correct amount or timing of gene expression lead to improper development and/or pathological conditions. Almost forty years after the first successful gene transfection in in vitro cell cultures, it is currently possible to regulate gene expression in an area-specific manner at any step of central nervous system development and in adulthood in experimental animals in vivo, even overcoming the very poor accessibility of the brain. Here, we will review the diverse approaches for acute gene transfer in vivo, highlighting their advantages and disadvantages with respect to the efficiency and specificity of transfection as well as to brain accessibility. In particular, we will present well-established chemical, physical and virus-based approaches suitable for different animal models, pointing out their current and future possible applications in basic and translational research as well as in gene therapy.
Keywords
shRNASonoporationTMCCNTEYFPmRNALRPHbdeGFPSVZCRISPR associated protein 9AAVintracerebroventriculargRNAICVPEIDcxSLNLNEHSVSV40Lipid nanoemulsionVSV-GMNPCoding DNACas9LPSCRISPRIUEEUEmHTTRVGAdVssDNAcDNASingle-stranded DNADNAiPSmessenger RNAshort hairpin RNAsmall-interfering RNAsiRNAAdenovirusesApoedeoxyribonucleic acidribonucleic acidRNAElectroporationintrathecalPolymersclustered regularly interspaced short palindromic repeatstumor necrosis factor alphaheparin-binding domainIn utero electroporationCNSdoublecortinguide RNAblood brain barrierBBBCMVcytomegaloviruscentral nervous systemTNF-αUltrasoundlipopolysaccharidesubventricular zoneMicro-RNAMiRNANanoparticlesSolid lipid nanoparticlesMagnetic nanoparticlesCarbon nanotubesmutant HuntingtinRepReplicationSimian virus 40human immunodeficiency virusHIVVirusesAdeno-associated virusesHerpes simplex virusesHelper virusrabies virus glycoproteinPAMAMenhanced yellow fluorescent proteinenhanced green fluorescent proteinLipoprotein receptor-related proteinPolyamidoaminePolyethyleniminepolyethylene glycolPEGDOPECaPEncapsulationTrimethylated chitosanvesicular stomatitis virus glycoprotein
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Authors
Andrzej W. Cwetsch, Bruno Pinto, Annalisa Savardi, Laura Cancedda,