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Patients develop behavioural disturbances and cognitive decline, a possible consequence of neuroinflammation and abnormal substrate accumulation. Interleukin (IL)\u20101\u03b2 and interleukin\u20101 receptor antagonist (IL\u20101Ra) expression were significantly increased in both murine models and human MPSIII patients. We identified pathogenic mechanisms of inflammasome activation, including that disease\u2010specific 2\u2010O\u2010sulphated heparan sulphate was essential for priming an IL\u20101\u03b2 response via the Toll\u2010like receptor 4 complex. However, mucopolysaccharidosis IIIA primary and secondary storage substrates, such as amyloid beta, were both required to activate the NLRP3 inflammasome and initiate IL\u20101\u03b2 secretion. IL\u20101 blockade in mucopolysaccharidosis IIIA mice using IL\u20101 receptor type 1 knockout or haematopoietic stem cell gene therapy over\u2010expressing IL\u20101Ra reduced gliosis and completely prevented behavioural phenotypes. In conclusion, we demonstrate that IL\u20101 drives neuroinflammation, behavioural abnormality and cognitive decline in mucopolysaccharidosis IIIA, highlighting haematopoietic stem cell gene therapy treatment with IL\u20101Ra as a potential neuronopathic lysosomal disease treatment.<\/jats:p>","DOI":"10.15252\/emmm.201911185","type":"journal-article","created":{"date-parts":[[2020,2,14]],"date-time":"2020-02-14T07:22:18Z","timestamp":1581664938000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":52,"title":["Haematopoietic stem cell gene therapy with IL\u20101Ra rescues cognitive loss in mucopolysaccharidosis IIIA"],"prefix":"10.1038","volume":"12","author":[{"given":"Helen","family":"Parker","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stuart M","family":"Ellison","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca J","family":"Holley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claire","family":"O'Leary","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aiyin","family":"Liao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jalal","family":"Asadi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily","family":"Glover","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arunabha","family":"Ghosh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon","family":"Jones","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiona L","family":"Wilkinson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2250-2381","authenticated-orcid":false,"given":"David","family":"Brough","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanuel","family":"Pinteaux","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Herv\u00e9","family":"Boutin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9708-1112","authenticated-orcid":false,"given":"Brian W","family":"Bigger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2020,2,14]]},"reference":[{"key":"BFEMMM201911185_CR1","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1111\/acel.12409","volume":"15","author":"E Aflaki","year":"2016","unstructured":"Aflaki E, Moaven N, Borger DK, Lopez G, Westbroek W, Chae JJ, Marugan J, Patnaik S, Maniwang E, Gonzalez AN et\u00a0al (2016) Lysosomal storage and impaired autophagy lead to inflammasome activation in Gaucher macrophages. 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