{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,12]],"date-time":"2026-05-12T22:19:16Z","timestamp":1778624356619,"version":"3.51.4"},"reference-count":17,"publisher":"The Royal Society","issue":"1433","license":[{"start":{"date-parts":[[2003,5,29]],"date-time":"2003-05-29T00:00:00Z","timestamp":1054166400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/royalsociety.org\/journals\/ethics-policies\/data-sharing-mining\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Phil. Trans. R. Soc. Lond. B"],"published-print":{"date-parts":[[2003,5,29]]},"abstract":"<jats:p>\n                    Lysosomal storage diseases comprise a group of about 40 disorders, which in most cases are due to the deficiency of a lysosomal enzyme. Since lysosomal enzymes are involved in the degradation of various compounds, the diseases can be further subdivided according to which pathway is affected. Thus, enzyme deficiencies in the degradation pathway of glycosaminoglycans cause mucopolysaccharidosis, and deficiencies affecting glycopeptides cause glycoproteinosis. In glycolipid storage diseases enzymes are deficient that are involved in the degradation of sphingolipids. Mouse models are available for most of these diseases, and some of these mouse models have been used to study the applicability of\n                    <jats:italic>in vivo<\/jats:italic>\n                    gene therapy. We review the rationale for gene therapy in lysosomal disorders and present data, in particular, about trials in an animal model of metachromatic leukodystrophy. The data of these trials are compared with those obtained with animal models of other lysosomal diseases.\n                  <\/jats:p>","DOI":"10.1098\/rstb.2003.1277","type":"journal-article","created":{"date-parts":[[2003,9,9]],"date-time":"2003-09-09T11:27:32Z","timestamp":1063106852000},"page":"921-925","source":"Crossref","is-referenced-by-count":17,"title":["Gene therapy: prospects for glycolipid storage diseases"],"prefix":"10.1098","volume":"358","author":[{"given":"Volkmar","family":"Gieselmann","sequence":"first","affiliation":[{"name":"Institut f\u00fcr Physiologische Chemie, Rheinische-Friedrich-Wilhelms Universit\u00e4t, Bonn 53115, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ulrich","family":"Matzner","sequence":"additional","affiliation":[{"name":"Institut f\u00fcr Physiologische Chemie, Rheinische-Friedrich-Wilhelms Universit\u00e4t, Bonn 53115, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Diana","family":"Klein","sequence":"additional","affiliation":[{"name":"Institut f\u00fcr Physiologische Chemie, Rheinische-Friedrich-Wilhelms Universit\u00e4t, Bonn 53115, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jan","family":"Eric Mansson","sequence":"additional","affiliation":[{"name":"Institute for Clinical Neuroscience, Sahlgren's University Hospital, M\u00f6lndal 43180, Sweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rudi","family":"D'Hooge","sequence":"additional","affiliation":[{"name":"Laboratory of Neurochemistry and Behaviour, Born Bunge Foundation, University of Antwerp, Antwerp 2610, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter D.","family":"DeDeyn","sequence":"additional","affiliation":[{"name":"Laboratory of Neurochemistry and Behaviour, Born Bunge Foundation, University of Antwerp, Antwerp 2610, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Renate","family":"L\u00fcllmann Rauch","sequence":"additional","affiliation":[{"name":"Anatomisches Institut, Christian Albrechts Universit\u00e4t, Kiel 24098, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dieter","family":"Hartmann","sequence":"additional","affiliation":[{"name":"Anatomisches Institut, Christian Albrechts Universit\u00e4t, Kiel 24098, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Klaus","family":"Harzer","sequence":"additional","affiliation":[{"name":"Institut f\u00fc r Hirnforschung, Eberhard\u2013Karls\u2013Universit\u00e4t, T\u00fcbingen 72070, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"175","published-online":{"date-parts":[[2003,5,29]]},"reference":[{"key":"p_1","doi-asserted-by":"publisher","DOI":"10.1038\/85454"},{"key":"p_2","doi-asserted-by":"publisher","DOI":"10.2174\/1566524023363112"},{"key":"p_3","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/8.5.831"},{"key":"p_4","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.93.25.14821"},{"key":"p_5","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.051634498"},{"key":"p_6","doi-asserted-by":"crossref","first-page":"986","DOI":"10.1182\/blood.V90.3.986","article-title":"Kinetics of central nervous system microglial and macrophage engraftment: analysis using a transgenic bone marrow transplantation model","volume":"90","author":"Kennedy D. 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Series B: Biological Sciences"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/royalsocietypublishing.org\/doi\/pdf\/10.1098\/rstb.2003.1277","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/royalsocietypublishing.org\/doi\/pdf\/10.1098\/rstb.2003.1277","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,12]],"date-time":"2026-05-12T21:22:35Z","timestamp":1778620955000},"score":1,"resource":{"primary":{"URL":"http:\/\/royalsocietypublishing.org\/rstb\/article\/358\/1433\/921-925\/20186"}},"subtitle":[],"editor":[{"given":"R. A.","family":"Dwek","sequence":"additional","affiliation":[],"role":[{"role":"editor","vocabulary":"crossref"}]},{"given":"T. D.","family":"Butters","sequence":"additional","affiliation":[],"role":[{"role":"editor","vocabulary":"crossref"}]},{"given":"F. M.","family":"Platt","sequence":"additional","affiliation":[],"role":[{"role":"editor","vocabulary":"crossref"}]},{"given":"T. M.","family":"Cox","sequence":"additional","affiliation":[],"role":[{"role":"editor","vocabulary":"crossref"}]}],"short-title":[],"issued":{"date-parts":[[2003,5,29]]},"references-count":17,"journal-issue":{"issue":"1433","published-print":{"date-parts":[[2003,5,29]]}},"alternative-id":["10.1098\/rstb.2003.1277"],"URL":"https:\/\/doi.org\/10.1098\/rstb.2003.1277","relation":{},"ISSN":["0962-8436","1471-2970"],"issn-type":[{"value":"0962-8436","type":"print"},{"value":"1471-2970","type":"electronic"}],"subject":[],"published":{"date-parts":[[2003,5,29]]}}}