{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T04:47:54Z","timestamp":1787028474446,"version":"build-2736575974"},"reference-count":69,"publisher":"MDPI AG","issue":"9","license":[{"start":{"date-parts":[[2022,4,27]],"date-time":"2022-04-27T00:00:00Z","timestamp":1651017600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Mucopolysaccharidosis type II (Hunter Syndrome) is a rare, x-linked recessive, progressive, multi-system, lysosomal storage disease caused by the deficiency of iduronate-2-sulfatase (IDS), which leads to the pathological storage of glycosaminoglycans in nearly all cell types, tissues and organs. The condition is clinically heterogeneous, and most patients present with a progressive, multi-system disease in their early years. This article outlines the pathology of the disorder and current treatment strategies, including a detailed review of haematopoietic stem cell transplant outcomes for MPSII. We then discuss haematopoietic stem cell gene therapy and how this can be employed for treatment of the disorder. We consider how preclinical innovations, including novel brain-targeted techniques, can be incorporated into stem cell gene therapy approaches to mitigate the neuropathological consequences of the condition.<\/jats:p>","DOI":"10.3390\/ijms23094854","type":"journal-article","created":{"date-parts":[[2022,4,27]],"date-time":"2022-04-27T22:20:20Z","timestamp":1651098020000},"page":"4854","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?"],"prefix":"10.3390","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6865-8509","authenticated-orcid":false,"given":"Claire","family":"Horgan","sequence":"first","affiliation":[{"name":"Blood and Marrow Transplant Unit, Department of Paediatric Haematology, Royal Manchester Children\u2019s Hospital, Manchester University NHS Foundation Trust, Manchester M13 9WL, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon A.","family":"Jones","sequence":"additional","affiliation":[{"name":"Willink Unit, Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Manchester M13 9WL, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9708-1112","authenticated-orcid":false,"given":"Brian W.","family":"Bigger","sequence":"additional","affiliation":[{"name":"Stem Cell and Neuropathies, Division of Cell Matrix Biology & Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Wynn","sequence":"additional","affiliation":[{"name":"Blood and Marrow Transplant Unit, Department of Paediatric Haematology, Royal Manchester Children\u2019s Hospital, Manchester University NHS Foundation Trust, Manchester M13 9WL, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2022,4,27]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1007\/s40265-019-01147-4","article-title":"Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments","volume":"79","author":"Sawamoto","year":"2019","journal-title":"Drugs"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"846","DOI":"10.1038\/sj.ejhg.5201623","article-title":"Genistein-mediated inhibition of glycosaminoglycan synthesis as a basis for gene expression-targeted isoflavone therapy for mucopolysaccharidoses","volume":"14","author":"Piotrowska","year":"2006","journal-title":"Eur. 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