{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T18:21:48Z","timestamp":1791397308702,"version":"4.3.3"},"reference-count":86,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2021,5,23]],"date-time":"2021-05-23T00:00:00Z","timestamp":1621728000000},"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 (MPS II) is a lysosomal storage disorder based on a mutation in the IDS gene that encodes iduronate 2-sulphatase. As a result, there is an accumulation of glycosaminoglycans\u2014heparan sulphate and dermatan sulphate\u2014in almost all body tissues, which leads to their dysfunction. Currently, the primary treatment is enzyme replacement therapy, which improves the course of the disease by reducing somatic symptoms, including hepatomegaly and splenomegaly. The enzyme, however, does not cross the blood\u2013brain barrier, and no improvement in the function of the central nervous system has been observed in patients with the severe form of the disease. An alternative method of treatment that solves typical problems of enzyme replacement therapy is gene therapy, i.e., delivery of the correct gene to target cells through an appropriate vector. Much progress has been made in applying gene therapy for MPS II, from cellular models to human clinical trials. In this article, we briefly present the history and basics of gene therapy and discuss the current state of knowledge about the methods of this therapy in mucopolysaccharidosis type II.<\/jats:p>","DOI":"10.3390\/ijms22115490","type":"journal-article","created":{"date-parts":[[2021,5,23]],"date-time":"2021-05-23T23:59:03Z","timestamp":1621814343000},"page":"5490","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":38,"title":["Gene Therapy for Mucopolysaccharidosis Type II\u2014A Review of the Current Possibilities"],"prefix":"10.3390","volume":"22","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9935-472X","authenticated-orcid":false,"given":"Pawe\u0142","family":"Zapolnik","sequence":"first","affiliation":[{"name":"Students\u2019 Scientific Association of Clinical Genetics, Department of Clinical Genetics, Medical College, University of Rzesz\u00f3w, 35-959 Rzesz\u00f3w, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antoni","family":"Pyrkosz","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics, Medical College, University of Rzesz\u00f3w, 35-959 Rzesz\u00f3w, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2021,5,23]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1111\/imj.15100","article-title":"Treatable lysosomal storage diseases in the advent of disease-specific therapy","volume":"50","author":"Peters","year":"2020","journal-title":"Int. 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