{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,16]],"date-time":"2026-03-16T23:26:20Z","timestamp":1773703580969,"version":"3.50.1"},"reference-count":126,"publisher":"MDPI AG","issue":"4","license":[{"start":{"date-parts":[[2020,4,2]],"date-time":"2020-04-02T00:00:00Z","timestamp":1585785600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"publisher","award":["BA815\/12-1"],"award-info":[{"award-number":["BA815\/12-1"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"publisher","award":["Funding Programme Open Access Publishing"],"award-info":[{"award-number":["Funding Programme Open Access Publishing"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>GM1-gangliosidosis is caused by a reduced activity of \u03b2-galactosidase (Glb1), resulting in intralysosomal accumulations of GM1. The aim of this study was to reveal the pathogenic mechanisms of GM1-gangliosidosis in a new Glb1 knockout mouse model. Glb1\u2212\/\u2212 mice were analyzed clinically, histologically, immunohistochemically, electrophysiologically and biochemically. Morphological lesions in the central nervous system were already observed in two-month-old mice, whereas functional deficits, including ataxia and tremor, did not start before 3.5-months of age. This was most likely due to a reduced membrane resistance as a compensatory mechanism. Swollen neurons exhibited intralysosomal storage of lipids extending into axons and amyloid precursor protein positive spheroids. Additionally, axons showed a higher kinesin and lower dynein immunoreactivity compared to wildtype controls. Glb1\u2212\/\u2212 mice also demonstrated loss of phosphorylated neurofilament positive axons and a mild increase in non-phosphorylated neurofilament positive axons. Moreover, marked astrogliosis and microgliosis were found, but no demyelination. In addition to the main storage material GM1, GA1, sphingomyelin, phosphatidylcholine and phosphatidylserine were elevated in the brain. In summary, the current Glb1\u2212\/\u2212 mice exhibit a so far undescribed axonopathy and a reduced membrane resistance to compensate the functional effects of structural changes. They can be used for detailed examinations of axon\u2013glial interactions and therapy trials of lysosomal storage diseases.<\/jats:p>","DOI":"10.3390\/jcm9041004","type":"journal-article","created":{"date-parts":[[2020,4,2]],"date-time":"2020-04-02T13:39:34Z","timestamp":1585834774000},"page":"1004","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["Axonopathy and Reduction of Membrane Resistance: Key Features in a New Murine Model of Human GM1-Gangliosidosis"],"prefix":"10.3390","volume":"9","author":[{"given":"Deborah","family":"Eikelberg","sequence":"first","affiliation":[{"name":"Department of Pathology, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Annika","family":"Lehmbecker","sequence":"additional","affiliation":[{"name":"Department of Pathology, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Graham","family":"Brogden","sequence":"additional","affiliation":[{"name":"Department of Physiological Chemistry, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Witchaya","family":"Tongtako","sequence":"additional","affiliation":[{"name":"Department of Pathology, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"},{"name":"c\/o Faculty of Veterinary Science, Prince of Sonkla University, 5 Karnjanavanich Rd., Hat Yai, Songkhla 90110, Thailand"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kerstin","family":"Hahn","sequence":"additional","affiliation":[{"name":"Department of Pathology, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andre","family":"Habierski","sequence":"additional","affiliation":[{"name":"Department of Pathology, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julia B.","family":"Hennermann","sequence":"additional","affiliation":[{"name":"Villa Metabolica, University of Mainz, Langenbeckstra\u00dfe 2, D-55131 Mainz, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4884-8425","authenticated-orcid":false,"given":"Hassan Y.","family":"Naim","sequence":"additional","affiliation":[{"name":"Department of Physiological Chemistry, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Felix","family":"Felmy","sequence":"additional","affiliation":[{"name":"Department for Physiology and Cell Biology, University of Veterinary Medicine Hannover, 30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wolfgang","family":"Baumg\u00e4rtner","sequence":"additional","affiliation":[{"name":"Department of Pathology, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7973-7405","authenticated-orcid":false,"given":"Ingo","family":"Gerhauser","sequence":"additional","affiliation":[{"name":"Department of Pathology, University of Veterinary Medicine Hannover, D-30559 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2020,4,2]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1002","DOI":"10.1126\/science.160.3831.1002","article-title":"Generalized Gangliosidosis: Beta-Galactosidase Deficiency","volume":"160","author":"Okada","year":"1968","journal-title":"Science"},{"key":"ref_2","first-page":"649","article-title":"Animal Model: Feline Gm1 Gangliosidosis","volume":"74","author":"Baker","year":"1974","journal-title":"Am. 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