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dc.contributor.authorHumphries, Peteren
dc.contributor.authorFarrar, Gwynethen
dc.date.accessioned2021-02-23T13:38:39Z
dc.date.available2021-02-23T13:38:39Z
dc.date.issued2020en
dc.date.submitted2020en
dc.identifier.citationMillington-Ward, S. and Chadderton, N. and Berkeley, M. and Finnegan, L.K. and Hanlon, K.S. and Carrigan, M. and Humphries, P. and Kenna, P.F. and Palfi, A. and Farrar, G.J., Novel 199 base pair NEFH promoter drives expression in retinal ganglion cells, Scientific Reports, 10, 1, 2020en
dc.identifier.otherYen
dc.identifier.urihttp://hdl.handle.net/2262/95314
dc.descriptionPUBLISHEDen
dc.descriptioncited By 0en
dc.description.abstractRetinal ganglion cells (RGCs) are known to be involved in several ocular disorders, including glaucoma and Leber hereditary optic neuropathy (LHON), and hence represent target cells for gene therapies directed towards these diseases. Restricting gene therapeutics to the target cell type in many situations may be preferable compared to ubiquitous transgene expression, stimulating researchers to identify RGC-specific promoters, particularly promoter sequences that may also be appropriate in size to fit readily into recombinant adeno associated viral (AAV) vectors, the vector of choice for many ocular gene therapies. In the current study we analysed EGFP expression driven by various sequences of the putative human NEFH promoter in order to define sequences required for preferential expression in RGCs. EGFP expression profiles from four different potential NEFH promoter constructs were compared in vivo in mice using retinal histology and mRNA expression analysis. Notably, two efficient promoter sequences, one comprising just 199 bp, are presented in the study.en
dc.language.isoenen
dc.relation.ispartofseriesScientific Reportsen
dc.relation.ispartofseries10en
dc.relation.ispartofseries1en
dc.rightsYen
dc.subjectGFP expression profilesen
dc.subjectRetinal ganglion cells (RGCs)en
dc.subjectglaucoma and Leber hereditary optic neuropathy (LHONen
dc.titleNovel 199 base pair NEFH promoter drives expression in retinal ganglion cellsen
dc.typeJournal Articleen
dc.type.supercollectionscholarly_publicationsen
dc.type.supercollectionrefereed_publicationsen
dc.identifier.peoplefinderurlhttp://people.tcd.ie/phumphrsen
dc.identifier.peoplefinderurlhttp://people.tcd.ie/gjfarraren
dc.identifier.rssinternalid224319en
dc.identifier.doihttp://dx.doi.org/10.1038/s41598-020-73257-zen
dc.rights.ecaccessrightsopenAccess
dc.contributor.sponsorGrantNumber16/1A/4452en


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