{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T22:33:54Z","timestamp":1761863634714},"reference-count":32,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2016,11,10]],"date-time":"2016-11-10T00:00:00Z","timestamp":1478736000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2016,11,10]],"date-time":"2016-11-10T00:00:00Z","timestamp":1478736000000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"funder":[{"name":"Shire (USA)"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2017,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Intra\u2010cerebrospinal fluid (CSF) injection of recombinant human lysosomal enzyme is a potential treatment strategy for several neurodegenerative lysosomal storage disorders including Sanfilippo syndrome (Mucopolysaccharidosis type IIIA; MPS IIIA). Here we have utilised the MPS IIIA Huntaway dog model to compare the effectiveness of the repeated intermittent bolus injection strategy being used in the trials with an alternate approach; slow, continual infusion of replacement enzyme (recombinant human sulphamidase; rhSGSH) into the spinal CSF using a SynchroMed II\u00ae pump attached to a spinal infusion cannula. The ability of each enzyme delivery strategy to ameliorate lesions in MPS IIIA brain was determined in animals treated from \u223cthree\u2010 to six\u2010months of age. Controls received buffer or no treatment. Significant reductions in heparan sulphate (primary substrate) were observed in brain samples from dogs treated via either cisternal or lumbar spinal CSF bolus injection methods and also in slow intra\u2010spinal CSF infusion\u2010treated dogs. The extent of the reduction differed regionally. Pump\u2010delivered rhSGSH was less effective in reducing secondary substrate (G<jats:sub>M3<\/jats:sub> ganglioside) in deeper aspects of cerebral cortex, and although near\u2010amelioration of microglial activation was seen in superficial (but not deep) layers of cerebral cortex in both bolus enzyme\u2010treated groups, pump\u2010infusion of rhSGSH had little impact on microgliosis. While continual low\u2010dose infusion of rhSGSH into MPS IIIA dog CSF reduces disease\u2010based lesions in brain, it was not as efficacious as repeated cisternal or spinal CSF bolus infusion of rhSGSH over the time\u2010frame of these experiments.<\/jats:p>","DOI":"10.1007\/s10545-016-9994-1","type":"journal-article","created":{"date-parts":[[2016,11,10]],"date-time":"2016-11-10T09:09:50Z","timestamp":1478768990000},"page":"443-453","update-policy":"http:\/\/dx.doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Slow, continuous enzyme replacement via spinal CSF in dogs with the paediatric\u2010onset neurodegenerative disease, MPS IIIA"],"prefix":"10.1002","volume":"40","author":[{"given":"Barbara","family":"King","sequence":"first","affiliation":[{"name":"Lysosomal Diseases Research Unit South Australian Health and Medical Research Institute PO Box 11060 Adelaide SA 5001 Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Neil R.","family":"Marshall","sequence":"additional","affiliation":[{"name":"Institute of Veterinary, Animal and Biomedical Science Massey University Palmerston North New Zealand"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sofia","family":"Hassiotis","sequence":"additional","affiliation":[{"name":"Lysosomal Diseases Research Unit South Australian Health and Medical Research Institute PO Box 11060 Adelaide SA 5001 Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul J.","family":"Trim","sequence":"additional","affiliation":[{"name":"Lysosomal Diseases Research Unit South Australian Health and Medical Research Institute PO Box 11060 Adelaide SA 5001 Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Justin","family":"Tucker","sequence":"additional","affiliation":[{"name":"Lysosomal Diseases Research Unit South Australian Health and Medical 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