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Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease

dc.contributor.authorSergijenko, Ana
dc.contributor.authorLangford-Smith, Alexander
dc.contributor.authorLiao, Ai Y
dc.contributor.authorPickford, Claire E
dc.contributor.authorMcDermott, John
dc.contributor.authorNowinski, Gabriel
dc.contributor.authorLangford-Smith, Kia J
dc.contributor.authorMerry, Catherine L R
dc.contributor.authorJones, Simon A
dc.contributor.authorWraith, J Edmond
dc.contributor.authorWynn, Robert F
dc.contributor.authorWilkinson, Fiona L
dc.contributor.authorBigger, Brian W
dc.date.accessioned2014-11-25T18:37:44Z
dc.date.available2014-11-25T18:37:44Z
dc.date.issued2013-10
dc.identifier44341132
dc.identifier291d7692-990b-4de6-8711-f0a15e934592
dc.identifier23748415
dc.identifier84885018977
dc.identifier.citationSergijenko , A , Langford-Smith , A , Liao , A Y , Pickford , C E , McDermott , J , Nowinski , G , Langford-Smith , K J , Merry , C L R , Jones , S A , Wraith , J E , Wynn , R F , Wilkinson , F L & Bigger , B W 2013 , ' Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease ' , Molecular therapy : the journal of the American Society of Gene Therapy , vol. 21 , no. 10 , pp. 1938-49 . https://doi.org/10.1038/mt.2013.141en
dc.identifier.issn1525-0024
dc.identifier.urihttp://hdl.handle.net/2164/4001
dc.format.extent12
dc.format.extent1521288
dc.language.isoeng
dc.relation.ispartofMolecular therapy : the journal of the American Society of Gene Therapyen
dc.subjectAnimalsen
dc.subjectAntigens, CD11ben
dc.subjectBrainen
dc.subjectCell Lineen
dc.subjectDisease Models, Animalen
dc.subjectFemaleen
dc.subjectGenetic Therapyen
dc.subjectGenetic Vectorsen
dc.subjectGreen Fluorescent Proteinsen
dc.subjectHematopoietic Stem Cell Transplantationen
dc.subjectHematopoietic Stem Cellsen
dc.subjectHeparitin Sulfateen
dc.subjectHumansen
dc.subjectHydrolasesen
dc.subjectLentivirusen
dc.subjectLeukocytesen
dc.subjectLysosomesen
dc.subjectMiceen
dc.subjectMice, Inbred C57BLen
dc.subjectMicrogliaen
dc.subjectMucopolysaccharidosis IIIen
dc.subjectMyeloid Cellsen
dc.subjectOrgan Specificityen
dc.subjectPromoter Regions, Geneticen
dc.subjectR Medicineen
dc.subject.lccRen
dc.titleMyeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal diseaseen
dc.typeJournal articleen
dc.contributor.institutionUniversity of Aberdeen.Applied Medicineen
dc.description.statusPeer revieweden
dc.identifier.doi10.1038/mt.2013.141


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