{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T01:03:49Z","timestamp":1773277429818,"version":"3.50.1"},"reference-count":27,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2020,10,30]],"date-time":"2020-10-30T00:00:00Z","timestamp":1604016000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Fondazione Istituto di Ricerca Pediatrica Citt\u00e0 della Speranza","award":["19\/05"],"award-info":[{"award-number":["19\/05"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Life"],"abstract":"<jats:p>Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder that occurs due to the deficit of the lysosomal enzyme iduronate 2-sulfatase (IDS) that leads to the storage of the glycosaminoglycan heparan- and dermatan-sulfate in all organs and tissues. It is characterized by important clinical features and the severe form presents with a heavy neurological involvement. However, almost nothing is known about the neuropathogenesis of MPS II. To address this issue, we developed a ubiquitous, neuronal, and glial-specific knockdown model in Drosophila melanogaster by using the RNA interference (RNAi) approach. Knockdown of the Ids\/CG12014 gene resulted in a significant reduction of the Ids gene expression and enzymatic activity. However, glycosaminoglycan storage, survival, molecular markers (Atg8a, Lamp1, Rab11), and locomotion behavior were not affected. Even strongly reduced, IDS-activity was enough to prevent a pathological phenotype in a MPS II RNAi fruit fly. Thus, a Drosophila MPS II model requires complete abolishment of the enzymatic activity.<\/jats:p>","DOI":"10.3390\/life10110263","type":"journal-article","created":{"date-parts":[[2020,10,30]],"date-time":"2020-10-30T00:21:06Z","timestamp":1604017266000},"page":"263","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Modeling Mucopolysaccharidosis Type II in the Fruit Fly by Using the RNA Interference Approach"],"prefix":"10.3390","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0233-1677","authenticated-orcid":false,"given":"Laura","family":"Rigon","sequence":"first","affiliation":[{"name":"Molecular Developmental Biology Unit, Life &amp; Medical Sciences Institute (LIMES), University of Bonn, Carl-Troll-Stra\u00dfe 31, 53115 Bonn, Germany"},{"name":"Fondazione Istituto di Ricerca Pediatrica \u201cCitt\u00e0 della Speranza\u201d, Corso Stati Uniti 4, 35127 Padova, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nicole","family":"Kucharowski","sequence":"additional","affiliation":[{"name":"Molecular Developmental Biology Unit, Life &amp; Medical Sciences Institute (LIMES), University of Bonn, Carl-Troll-Stra\u00dfe 31, 53115 Bonn, Germany"},{"name":"Neuronal Wiring, Life &amp; Medical Sciences Institute (LIMES), University of Bonn, Carl-Troll-Stra\u00dfe 31, 53115 Bonn, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Franka","family":"Eckardt","sequence":"additional","affiliation":[{"name":"Molecular Developmental Biology Unit, Life &amp; Medical Sciences Institute (LIMES), University of Bonn, Carl-Troll-Stra\u00dfe 31, 53115 Bonn, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6352-7198","authenticated-orcid":false,"given":"Reinhard","family":"Bauer","sequence":"additional","affiliation":[{"name":"Molecular Developmental Biology Unit, Life &amp; Medical Sciences Institute (LIMES), University of Bonn, Carl-Troll-Stra\u00dfe 31, 53115 Bonn, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2020,10,30]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1016\/j.ymgme.2017.05.016","article-title":"Epidemiology of mucopolysaccharidoses","volume":"121","author":"Khan","year":"2017","journal-title":"Mol. 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