{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T17:39:55Z","timestamp":1791221995803,"version":"4.1.0"},"reference-count":53,"publisher":"MDPI AG","issue":"10","license":[{"start":{"date-parts":[[2023,5,22]],"date-time":"2023-05-22T00:00:00Z","timestamp":1684713600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100006769","name":"Russian Science Foundation","doi-asserted-by":"publisher","award":["22-25-00721"],"award-info":[{"award-number":["22-25-00721"]}],"id":[{"id":"10.13039\/501100006769","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Mutations in the GBA1 gene, encoding the lysosomal enzyme glucocerebrosidase (GCase), cause Gaucher disease (GD) and are the most common genetic risk factor for Parkinson\u2019s disease (PD). Pharmacological chaperones (PCs) are being developed as an alternative treatment approach for GD and PD. To date, NCGC00241607 (NCGC607) is one of the most promising PCs. Using molecular docking and molecular dynamics simulation we identified and characterized six allosteric binding sites on the GCase surface suitable for PCs. Two sites were energetically more preferable for NCGC607 and located nearby to the active site of the enzyme. We evaluated the effects of NCGC607 treatment on GCase activity and protein levels, glycolipids concentration in cultured macrophages from GD (n = 9) and GBA-PD (n = 5) patients as well as in induced human pluripotent stem cells (iPSC)\u2014derived dopaminergic (DA) neurons from GBA-PD patient. The results showed that NCGC607 treatment increased GCase activity (by 1.3-fold) and protein levels (by 1.5-fold), decreased glycolipids concentration (by 4.0-fold) in cultured macrophages derived from GD patients and also enhanced GCase activity (by 1.5-fold) in cultured macrophages derived from GBA-PD patients with N370S mutation (p &lt; 0.05). In iPSC-derived DA neurons from GBA-PD patients with N370S mutation NCGC607 treatment increased GCase activity and protein levels by 1.1-fold and 1.7-fold (p &lt; 0.05). Thus, our results showed that NCGC607 could bind to allosteric sites on the GCase surface and confirmed its efficacy on cultured macrophages from GD and GBA-PD patients as well as on iPSC-derived DA neurons from GBA-PD patients.<\/jats:p>","DOI":"10.3390\/ijms24109105","type":"journal-article","created":{"date-parts":[[2023,5,23]],"date-time":"2023-05-23T03:20:06Z","timestamp":1684812006000},"page":"9105","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Potential Binding Sites of Pharmacological Chaperone NCGC00241607 on Mutant \u03b2-Glucocerebrosidase and Its Efficacy on Patient-Derived Cell Cultures in Gaucher and Parkinson\u2019s Disease"],"prefix":"10.3390","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6557-0253","authenticated-orcid":false,"given":"Alena E.","family":"Kopytova","sequence":"first","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"},{"name":"Department of Molecular Genetic and Nanobiological Technologies, Pavlov First Saint-Petersburg State Medical University, Saint-Petersburg 197022, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2767-9175","authenticated-orcid":false,"given":"George N.","family":"Rychkov","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"},{"name":"Institute of Biomedical Systems and Biotechnology, Peter the Great St.Petersburg Polytechnic University, Saint-Petersburg 195251, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander A.","family":"Cheblokov","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena V.","family":"Grigor\u2019eva","sequence":"additional","affiliation":[{"name":"Institute of Cytology and Genetics Siberian Branch of Russian Academy of Sciences, Novosibirsk 630090, Russia"},{"name":"Meshalkin National Medical Research Center, Ministry of Health of the Russian Federation, Novosibirsk 630055, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1952-4678","authenticated-orcid":false,"given":"Mikhail A.","family":"Nikolaev","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"},{"name":"Department of Molecular Genetic and Nanobiological Technologies, Pavlov First Saint-Petersburg State Medical University, Saint-Petersburg 197022, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena S.","family":"Yarkova","sequence":"additional","affiliation":[{"name":"Institute of Cytology and Genetics Siberian Branch of Russian Academy of Sciences, Novosibirsk 630090, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana A.","family":"Sorogina","sequence":"additional","affiliation":[{"name":"Institute of Cytology and Genetics Siberian Branch of Russian Academy of Sciences, Novosibirsk 630090, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Farid M.","family":"Ibatullin","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Galina V.","family":"Baydakova","sequence":"additional","affiliation":[{"name":"Research Centre for Medical Genetics, Moscow 115522, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2919-1878","authenticated-orcid":false,"given":"Artem D.","family":"Izyumchenko","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"},{"name":"Department of Molecular Genetic and Nanobiological Technologies, Pavlov First Saint-Petersburg State Medical University, Saint-Petersburg 197022, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daria A.","family":"Bogdanova","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4857-2046","authenticated-orcid":false,"given":"Vitali M.","family":"Boitsov","sequence":"additional","affiliation":[{"name":"Laboratory of Nanobiotechnology, Saint-Petersburg National Research Academic University of the Russian Academy of Sciences, Saint-Petersburg 194021, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akim V.","family":"Rybakov","sequence":"additional","affiliation":[{"name":"N.P. Bechtereva Institute of the Human Brain RAS, Saint-Petersburg 197376, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina V.","family":"Miliukhina","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"},{"name":"N.P. Bechtereva Institute of the Human Brain RAS, Saint-Petersburg 197376, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vadim A.","family":"Bezrukikh","sequence":"additional","affiliation":[{"name":"Almazov National Medical Research Centre, Saint-Petersburg 197341, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Galina N.","family":"Salogub","sequence":"additional","affiliation":[{"name":"Almazov National Medical Research Centre, Saint-Petersburg 197341, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ekaterina Y.","family":"Zakharova","sequence":"additional","affiliation":[{"name":"Research Centre for Medical Genetics, Moscow 115522, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sofya N.","family":"Pchelina","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"},{"name":"Department of Molecular Genetic and Nanobiological Technologies, Pavlov First Saint-Petersburg State Medical University, Saint-Petersburg 197022, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anton K.","family":"Emelyanov","sequence":"additional","affiliation":[{"name":"Petersburg Nuclear Physics Institute Named by B.P. Konstantinov of National Research Center \u00abKurchatov Institute\u00bb, Gatchina 188300, Russia"},{"name":"Department of Molecular Genetic and Nanobiological Technologies, Pavlov First Saint-Petersburg State Medical University, Saint-Petersburg 197022, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,5,22]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1186\/s13024-019-0336-2","article-title":"Glucocerebrosidase and Its Relevance to Parkinson Disease","volume":"14","author":"Do","year":"2019","journal-title":"Mol. Neurodegener."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1111\/j.0009-9163.2004.00217.x","article-title":"Gaucher\u2019s Disease: A Paradigm for Interventional Genetics","volume":"65","author":"Germain","year":"2004","journal-title":"Clin. Genet."},{"key":"ref_3","unstructured":"The Human Gene Mutation Database (2023, May 19). 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