Sergijenko, AnaLangford-Smith, AlexanderLiao, Ai YPickford, Claire EMcDermott, JohnNowinski, GabrielLangford-Smith, Kia JMerry, Catherine L RJones, Simon AWraith, J EdmondWynn, Robert FWilkinson, Fiona LBigger, Brian W2014-11-252014-11-252013-10Sergijenko, 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.2013.1411525-0024http://hdl.handle.net/2164/4001121521288engAnimalsAntigens, CD11bBrainCell LineDisease Models, AnimalFemaleGenetic TherapyGenetic VectorsGreen Fluorescent ProteinsHematopoietic Stem Cell TransplantationHematopoietic Stem CellsHeparitin SulfateHumansHydrolasesLentivirusLeukocytesLysosomesMiceMice, Inbred C57BLMicrogliaMucopolysaccharidosis IIIMyeloid CellsOrgan SpecificityPromoter Regions, GeneticR MedicineRMyeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal diseaseJournal article10.1038/mt.2013.1412110