{"id":"https://openalex.org/W7166589718","doi":"https://doi.org/10.64898/2026.06.24.734200","title":"Effects of bromodomain protein inhibition in a mouse model of Niemann-Pick type C disease","display_name":"Effects of bromodomain protein inhibition in a mouse model of Niemann-Pick type C disease","publication_year":2026,"publication_date":"2026-06-29","ids":{"openalex":"https://openalex.org/W7166589718","doi":"https://doi.org/10.64898/2026.06.24.734200"},"language":"en","primary_location":{"id":"doi:10.64898/2026.06.24.734200","doi":"https://doi.org/10.64898/2026.06.24.734200","is_oa":true,"landing_page_url":"https://doi.org/10.64898/2026.06.24.734200","pdf_url":null,"source":{"id":"https://openalex.org/S4306402567","display_name":"bioRxiv (Cold Spring Harbor Laboratory)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2750212522","host_organization_name":"Cold Spring Harbor Laboratory","host_organization_lineage":["https://openalex.org/I2750212522"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"bioRxiv","raw_type":"posted-content"},"volume":null,"type":"preprint","is_traditional":true,"indexed_in":["crossref"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://doi.org/10.64898/2026.06.24.734200","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5058579947","display_name":"Martina Parente","orcid":"https://orcid.org/0000-0002-9135-567X","observed_orcids":["https://orcid.org/0000-0002-9135-567X"]},"institutions":[{"id":"https://openalex.org/I119003972","display_name":"Roma Tre University","ror":"https://ror.org/05vf0dg29","country_code":"IT","type":"education","lineage":["https://openalex.org/I119003972"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Martina Parente","raw_affiliation_strings":["Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy"],"raw_orcid":"https://orcid.org/0000-0002-9135-567X","affiliations":[{"raw_affiliation_string":"Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy","institution_ids":["https://openalex.org/I119003972"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5074546915","display_name":"Am\u00e9lie Barthelemy","orcid":"https://orcid.org/0009-0008-5113-5876","observed_orcids":["https://orcid.org/0009-0008-5113-5876"]},"institutions":[{"id":"https://openalex.org/I1294671590","display_name":"Centre National de la Recherche Scientifique","ror":"https://ror.org/02feahw73","country_code":"FR","type":"government","lineage":["https://openalex.org/I1294671590"]},{"id":"https://openalex.org/I4210117903","display_name":"Institut des Neurosciences Cellulaires et Int\u00e9gratives","ror":"https://ror.org/025mhd687","country_code":"FR","type":"facility","lineage":["https://openalex.org/I1294671590","https://openalex.org/I4210096427","https://openalex.org/I4210117903","https://openalex.org/I68947357"]},{"id":"https://openalex.org/I68947357","display_name":"Universit\u00e9 de Strasbourg","ror":"https://ror.org/00pg6eq24","country_code":"FR","type":"education","lineage":["https://openalex.org/I68947357"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Am\u00e9lie Barthelemy","raw_affiliation_strings":["Centre National de la Recherche Scientifique, Universit\u00e9 de Strasbourg, Institut des Neurosciences Cellulaires et Int\u00e9gratives, 8 all\u00e9e du G\u00e9n\u00e9ral Rouvillois, 67000 Strasbourg, France"],"raw_orcid":"https://orcid.org/0009-0008-5113-5876","affiliations":[{"raw_affiliation_string":"Centre National de la Recherche Scientifique, Universit\u00e9 de Strasbourg, Institut des Neurosciences Cellulaires et Int\u00e9gratives, 8 all\u00e9e du G\u00e9n\u00e9ral Rouvillois, 67000 Strasbourg, France","institution_ids":["https://openalex.org/I1294671590","https://openalex.org/I4210117903","https://openalex.org/I68947357"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5043534105","display_name":"Sara Caputo","orcid":"https://orcid.org/0009-0009-9939-3767","observed_orcids":["https://orcid.org/0009-0009-9939-3767"]},"institutions":[{"id":"https://openalex.org/I119003972","display_name":"Roma Tre University","ror":"https://ror.org/05vf0dg29","country_code":"IT","type":"education","lineage":["https://openalex.org/I119003972"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Sara Caputo","raw_affiliation_strings":["Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy"],"raw_orcid":"https://orcid.org/0009-0009-9939-3767","affiliations":[{"raw_affiliation_string":"Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy","institution_ids":["https://openalex.org/I119003972"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5139564364","display_name":"Noemie Charlery-Adele","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1294671590","display_name":"Centre National de la Recherche Scientifique","ror":"https://ror.org/02feahw73","country_code":"FR","type":"government","lineage":["https://openalex.org/I1294671590"]},{"id":"https://openalex.org/I4387152384","display_name":"Chronobiotron","ror":"https://ror.org/026fpwg41","country_code":"FR","type":"facility","lineage":["https://openalex.org/I1294671590","https://openalex.org/I4387152384","https://openalex.org/I68947357"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"No\u00e9mie Charlery-Ad\u00e8le","raw_affiliation_strings":["Chronobiotron, CNRS, UAR 3415, 67000 Strasbourg, France"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Chronobiotron, CNRS, UAR 3415, 67000 Strasbourg, France","institution_ids":["https://openalex.org/I1294671590","https://openalex.org/I4387152384"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5117718583","display_name":"Christian Galmiche","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1294671590","display_name":"Centre National de la Recherche Scientifique","ror":"https://ror.org/02feahw73","country_code":"FR","type":"government","lineage":["https://openalex.org/I1294671590"]},{"id":"https://openalex.org/I4387152384","display_name":"Chronobiotron","ror":"https://ror.org/026fpwg41","country_code":"FR","type":"facility","lineage":["https://openalex.org/I1294671590","https://openalex.org/I4387152384","https://openalex.org/I68947357"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Christian Galmiche","raw_affiliation_strings":["Chronobiotron, CNRS, UAR 3415, 67000 Strasbourg, France"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Chronobiotron, CNRS, UAR 3415, 67000 Strasbourg, France","institution_ids":["https://openalex.org/I1294671590","https://openalex.org/I4387152384"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5026161094","display_name":"Claudia Tonini","orcid":"https://orcid.org/0000-0002-9979-1142","observed_orcids":["https://orcid.org/0000-0002-9979-1142"]},"institutions":[{"id":"https://openalex.org/I119003972","display_name":"Roma Tre University","ror":"https://ror.org/05vf0dg29","country_code":"IT","type":"education","lineage":["https://openalex.org/I119003972"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Claudia Tonini","raw_affiliation_strings":["Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy"],"raw_orcid":"https://orcid.org/0000-0002-9979-1142","affiliations":[{"raw_affiliation_string":"Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy","institution_ids":["https://openalex.org/I119003972"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5010981951","display_name":"Simona Lobasso","orcid":"https://orcid.org/0000-0003-3566-4547","observed_orcids":["https://orcid.org/0000-0003-3566-4547"]},"institutions":[{"id":"https://openalex.org/I5561750","display_name":"University of Bari Aldo Moro","ror":"https://ror.org/027ynra39","country_code":"IT","type":"education","lineage":["https://openalex.org/I5561750"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Simona Lobasso","raw_affiliation_strings":["Department of Translational Biomedicine and Neuroscience, University of Bari Aldo Moro, piazza G. Cesare 11, 70124 Bari, Italy"],"raw_orcid":"https://orcid.org/0000-0003-3566-4547","affiliations":[{"raw_affiliation_string":"Department of Translational Biomedicine and Neuroscience, University of Bari Aldo Moro, piazza G. Cesare 11, 70124 Bari, Italy","institution_ids":["https://openalex.org/I5561750"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5099590225","display_name":"Danilo Prtvar","orcid":"https://orcid.org/0009-0004-8251-1441","observed_orcids":["https://orcid.org/0009-0004-8251-1441"]},"institutions":[{"id":"https://openalex.org/I2801533059","display_name":"German Center for Neurodegenerative Diseases","ror":"https://ror.org/043j0f473","country_code":"DE","type":"facility","lineage":["https://openalex.org/I1305996414","https://openalex.org/I2801533059","https://openalex.org/I4405256427"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Danilo Prtvar","raw_affiliation_strings":["German Center for Neurodegenerative Diseases (DZNE) Munich, 81377 Munich, Germany"],"raw_orcid":"https://orcid.org/0009-0004-8251-1441","affiliations":[{"raw_affiliation_string":"German Center for Neurodegenerative Diseases (DZNE) Munich, 81377 Munich, Germany","institution_ids":["https://openalex.org/I2801533059"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048884366","display_name":"Sabina Tahirovi\u0107","orcid":"https://orcid.org/0000-0003-4403-9559","observed_orcids":["https://orcid.org/0000-0003-4403-9559"]},"institutions":[{"id":"https://openalex.org/I2801533059","display_name":"German Center for Neurodegenerative Diseases","ror":"https://ror.org/043j0f473","country_code":"DE","type":"facility","lineage":["https://openalex.org/I1305996414","https://openalex.org/I2801533059","https://openalex.org/I4405256427"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Sabina Tahirovic","raw_affiliation_strings":["German Center for Neurodegenerative Diseases (DZNE) Munich, 81377 Munich, Germany"],"raw_orcid":"https://orcid.org/0000-0003-4403-9559","affiliations":[{"raw_affiliation_string":"German Center for Neurodegenerative Diseases (DZNE) Munich, 81377 Munich, Germany","institution_ids":["https://openalex.org/I2801533059"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5160270449","display_name":"Sophie Reibel","orcid":"https://orcid.org/0009-0007-0144-9351","observed_orcids":["https://orcid.org/0009-0007-0144-9351"]},"institutions":[{"id":"https://openalex.org/I1294671590","display_name":"Centre National de la Recherche Scientifique","ror":"https://ror.org/02feahw73","country_code":"FR","type":"government","lineage":["https://openalex.org/I1294671590"]},{"id":"https://openalex.org/I4387152384","display_name":"Chronobiotron","ror":"https://ror.org/026fpwg41","country_code":"FR","type":"facility","lineage":["https://openalex.org/I1294671590","https://openalex.org/I4387152384","https://openalex.org/I68947357"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Sophie Reibel Foisset","raw_affiliation_strings":["Chronobiotron, CNRS, UAR 3415, 67000 Strasbourg, France"],"raw_orcid":"https://orcid.org/0009-0007-0144-9351","affiliations":[{"raw_affiliation_string":"Chronobiotron, CNRS, UAR 3415, 67000 Strasbourg, France","institution_ids":["https://openalex.org/I1294671590","https://openalex.org/I4387152384"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5052144876","display_name":"Frank W. Pfrieger","orcid":"https://orcid.org/0000-0001-7085-1431","observed_orcids":["https://orcid.org/0000-0001-7085-1431","https://orcid.org/0000-0003-2511-6168"]},"institutions":[{"id":"https://openalex.org/I119003972","display_name":"Roma Tre University","ror":"https://ror.org/05vf0dg29","country_code":"IT","type":"education","lineage":["https://openalex.org/I119003972"]},{"id":"https://openalex.org/I4210150112","display_name":"Fondazione Santa Lucia","ror":"https://ror.org/05rcxtd95","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I4210150112","https://openalex.org/I4210153126"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Valentina Pallottini","raw_affiliation_strings":["Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy","Neuroendocrinology, Metabolism and Neuropharmacology Unit, LIFE Institute for Research and Health Care - Santa Lucia IRCCS, Via del Fosso Fiorano 64, 00143 Rome, Italy"],"raw_orcid":"https://orcid.org/0000-0003-2511-6168","affiliations":[{"raw_affiliation_string":"Department of Science, Section Biomedical Science and Technology, University Roma Tre, Viale Marconi 446, 00146 Rome, Italy","institution_ids":["https://openalex.org/I119003972"]},{"raw_affiliation_string":"Neuroendocrinology, Metabolism and Neuropharmacology Unit, LIFE Institute for Research and Health Care - Santa Lucia IRCCS, Via del Fosso Fiorano 64, 00143 Rome, Italy","institution_ids":["https://openalex.org/I4210150112"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5052144876","display_name":"Frank W. Pfrieger","orcid":"https://orcid.org/0000-0001-7085-1431","observed_orcids":["https://orcid.org/0000-0001-7085-1431","https://orcid.org/0000-0003-2511-6168"]},"institutions":[{"id":"https://openalex.org/I1294671590","display_name":"Centre National de la Recherche Scientifique","ror":"https://ror.org/02feahw73","country_code":"FR","type":"government","lineage":["https://openalex.org/I1294671590"]},{"id":"https://openalex.org/I4210117903","display_name":"Institut des Neurosciences Cellulaires et Int\u00e9gratives","ror":"https://ror.org/025mhd687","country_code":"FR","type":"facility","lineage":["https://openalex.org/I1294671590","https://openalex.org/I4210096427","https://openalex.org/I4210117903","https://openalex.org/I68947357"]},{"id":"https://openalex.org/I68947357","display_name":"Universit\u00e9 de Strasbourg","ror":"https://ror.org/00pg6eq24","country_code":"FR","type":"education","lineage":["https://openalex.org/I68947357"]}],"countries":["FR"],"is_corresponding":true,"raw_author_name":"Frank W. Pfrieger","raw_affiliation_strings":["Centre National de la Recherche Scientifique, Universit\u00e9 de Strasbourg, Institut des Neurosciences Cellulaires et Int\u00e9gratives, 8 all\u00e9e du G\u00e9n\u00e9ral Rouvillois, 67000 Strasbourg, France"],"raw_orcid":"https://orcid.org/0000-0001-7085-1431","affiliations":[{"raw_affiliation_string":"Centre National de la Recherche Scientifique, Universit\u00e9 de Strasbourg, Institut des Neurosciences Cellulaires et Int\u00e9gratives, 8 all\u00e9e du G\u00e9n\u00e9ral Rouvillois, 67000 Strasbourg, France","institution_ids":["https://openalex.org/I1294671590","https://openalex.org/I4210117903","https://openalex.org/I68947357"]}]}],"institutions":[],"countries_distinct_count":3,"institutions_distinct_count":8,"corresponding_author_ids":["https://openalex.org/A5052144876"],"corresponding_institution_ids":["https://openalex.org/I1294671590","https://openalex.org/I4210117903","https://openalex.org/I68947357"],"apc_list":null,"apc_paid":null,"fwci":null,"has_fulltext":false,"cited_by_count":0,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.9990000128746033,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.9990000128746033,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T12604","display_name":"Glycogen Storage Diseases and Myoclonus","score":9.999999747378752e-05,"subfield":{"id":"https://openalex.org/subfields/1303","display_name":"Biochemistry"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T14415","display_name":"Biomedical Research and Pathophysiology","score":9.999999747378752e-05,"subfield":{"id":"https://openalex.org/subfields/2727","display_name":"Nephrology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/niemann-pick-disease-type-c","display_name":"Niemann-Pick disease type C","score":0.996999979019165},{"id":"https://openalex.org/keywords/bromodomain-proteins","display_name":"bromodomain proteins","score":0.8059999942779541},{"id":"https://openalex.org/keywords/bet-inhibition","display_name":"BET inhibition","score":0.722000002861023},{"id":"https://openalex.org/keywords/jq1","display_name":"JQ1","score":0.9919999837875366},{"id":"https://openalex.org/keywords/cholesterol-transport","display_name":"cholesterol transport","score":0.6819999814033508},{"id":"https://openalex.org/keywords/sex-differences","display_name":"sex differences","score":0.7910000085830688},{"id":"https://openalex.org/keywords/mouse-model","display_name":"mouse model","score":0.7170000076293945},{"id":"https://openalex.org/keywords/histone-acetylation","display_name":"histone acetylation","score":0.753000020980835},{"id":"https://openalex.org/keywords/hepatic-cholesterol","display_name":"liver cholesterol","score":0.5640000104904175},{"id":"https://openalex.org/keywords/motor-coordination","display_name":"motor coordination","score":0.8859999775886536}],"concepts":[{"id":"https://openalex.org/C74401373","wikidata":"https://www.wikidata.org/wiki/Q1829843","display_name":"Bromodomain","level":4,"score":0.9398000240325928},{"id":"https://openalex.org/C79879829","wikidata":"https://www.wikidata.org/wiki/Q5571762","display_name":"Intracellular","level":2,"score":0.6262999773025513},{"id":"https://openalex.org/C149011108","wikidata":"https://www.wikidata.org/wiki/Q652985","display_name":"Transporter","level":3,"score":0.5608999729156494},{"id":"https://openalex.org/C203522944","wikidata":"https://www.wikidata.org/wiki/Q288322","display_name":"Autophagy","level":3,"score":0.5271999835968018},{"id":"https://openalex.org/C2779634854","wikidata":"https://www.wikidata.org/wiki/Q18037206","display_name":"BRD4","level":5,"score":0.5138999819755554},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.511900007724762},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.453000009059906},{"id":"https://openalex.org/C2780862049","wikidata":"https://www.wikidata.org/wiki/Q25326844","display_name":"BET inhibitor","level":5,"score":0.44350001215934753},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.43320000171661377},{"id":"https://openalex.org/C2778163477","wikidata":"https://www.wikidata.org/wiki/Q43656","display_name":"Cholesterol","level":2,"score":0.4287000000476837},{"id":"https://openalex.org/C11804247","wikidata":"https://www.wikidata.org/wiki/Q896177","display_name":"Protein\u2013protein interaction","level":2,"score":0.390500009059906},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.35269999504089355},{"id":"https://openalex.org/C185592680","wikidata":"https://www.wikidata.org/wiki/Q2329","display_name":"Chemistry","level":0,"score":0.3431999981403351},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.33250001072883606},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.32109999656677246},{"id":"https://openalex.org/C36503486","wikidata":"https://www.wikidata.org/wiki/Q11235244","display_name":"Domain (mathematical analysis)","level":2,"score":0.30799999833106995},{"id":"https://openalex.org/C57581600","wikidata":"https://www.wikidata.org/wiki/Q2449730","display_name":"Transport protein","level":2,"score":0.3073999881744385},{"id":"https://openalex.org/C127561419","wikidata":"https://www.wikidata.org/wiki/Q224527","display_name":"Downregulation and upregulation","level":3,"score":0.29580000042915344},{"id":"https://openalex.org/C74187038","wikidata":"https://www.wikidata.org/wiki/Q1418791","display_name":"Drug discovery","level":2,"score":0.2786000072956085},{"id":"https://openalex.org/C2780035454","wikidata":"https://www.wikidata.org/wiki/Q8386","display_name":"Drug","level":2,"score":0.2694000005722046},{"id":"https://openalex.org/C25602115","wikidata":"https://www.wikidata.org/wiki/Q407111","display_name":"Ubiquitin","level":3,"score":0.2619999945163727},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.2619999945163727},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.257099986076355},{"id":"https://openalex.org/C171897839","wikidata":"https://www.wikidata.org/wiki/Q417841","display_name":"Protein family","level":3,"score":0.2563000023365021},{"id":"https://openalex.org/C2780600689","wikidata":"https://www.wikidata.org/wiki/Q14864821","display_name":"EGR1","level":4,"score":0.25380000472068787},{"id":"https://openalex.org/C51639874","wikidata":"https://www.wikidata.org/wiki/Q167149","display_name":"Plasma protein binding","level":2,"score":0.25200000405311584},{"id":"https://openalex.org/C54009773","wikidata":"https://www.wikidata.org/wiki/Q1429031","display_name":"Transfection","level":3,"score":0.2515000104904175},{"id":"https://openalex.org/C179185449","wikidata":"https://www.wikidata.org/wiki/Q219699","display_name":"Suppressor","level":3,"score":0.250900000333786}],"mesh":[],"locations_count":2,"locations":[{"id":"doi:10.64898/2026.06.24.734200","doi":"https://doi.org/10.64898/2026.06.24.734200","is_oa":true,"landing_page_url":"https://doi.org/10.64898/2026.06.24.734200","pdf_url":null,"source":{"id":"https://openalex.org/S4306402567","display_name":"bioRxiv (Cold Spring Harbor Laboratory)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2750212522","host_organization_name":"Cold Spring Harbor Laboratory","host_organization_lineage":["https://openalex.org/I2750212522"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"bioRxiv","raw_type":"posted-content"},{"id":"pmh:oai:HAL:hal-05686860v1","doi":null,"is_oa":true,"landing_page_url":"https://hal.science/hal-05686860v1/document","pdf_url":null,"source":{"id":"https://openalex.org/S4306402512","display_name":"HAL","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I4405252976","host_organization_name":"Centre pour la Communication Scientifique Directe","host_organization_lineage":["https://openalex.org/I4405252976"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"2026","raw_type":"info:eu-repo/semantics/preprint"}],"best_oa_location":{"id":"doi:10.64898/2026.06.24.734200","doi":"https://doi.org/10.64898/2026.06.24.734200","is_oa":true,"landing_page_url":"https://doi.org/10.64898/2026.06.24.734200","pdf_url":null,"source":{"id":"https://openalex.org/S4306402567","display_name":"bioRxiv (Cold Spring Harbor Laboratory)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2750212522","host_organization_name":"Cold Spring Harbor Laboratory","host_organization_lineage":["https://openalex.org/I2750212522"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"bioRxiv","raw_type":"posted-content"},"sustainable_development_goals":[{"score":0.9537000060081482,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":115,"referenced_works":["https://openalex.org/W1623462303","https://openalex.org/W1827118256","https://openalex.org/W1966378611","https://openalex.org/W1967363711","https://openalex.org/W1967516512","https://openalex.org/W1976252335","https://openalex.org/W1978837079","https://openalex.org/W1981615019","https://openalex.org/W1983606596","https://openalex.org/W2003872705","https://openalex.org/W2008762509","https://openalex.org/W2011555635","https://openalex.org/W2012149266","https://openalex.org/W2029397111","https://openalex.org/W2037942173","https://openalex.org/W2041535798","https://openalex.org/W2045625322","https://openalex.org/W2045963276","https://openalex.org/W2052203413","https://openalex.org/W2053203314","https://openalex.org/W2059319138","https://openalex.org/W2063283156","https://openalex.org/W2065408118","https://openalex.org/W2065773869","https://openalex.org/W2068262899","https://openalex.org/W2070053386","https://openalex.org/W2076246718","https://openalex.org/W2081027701","https://openalex.org/W2088021663","https://openalex.org/W2096221379","https://openalex.org/W2103009833","https://openalex.org/W2109931172","https://openalex.org/W2111084948","https://openalex.org/W2113003930","https://openalex.org/W2115716807","https://openalex.org/W2117956230","https://openalex.org/W2120572492","https://openalex.org/W2127350458","https://openalex.org/W2134893181","https://openalex.org/W2138786573","https://openalex.org/W2139615060","https://openalex.org/W2147612731","https://openalex.org/W2150904359","https://openalex.org/W2155503300","https://openalex.org/W2160604070","https://openalex.org/W2164558617","https://openalex.org/W2168016965","https://openalex.org/W2320064004","https://openalex.org/W2345076448","https://openalex.org/W2511455883","https://openalex.org/W2513570588","https://openalex.org/W2550502192","https://openalex.org/W2562877463","https://openalex.org/W2587144250","https://openalex.org/W2614434863","https://openalex.org/W2765600203","https://openalex.org/W2768531312","https://openalex.org/W2789111316","https://openalex.org/W2805556481","https://openalex.org/W2889615530","https://openalex.org/W2911749820","https://openalex.org/W2949962845","https://openalex.org/W2952120501","https://openalex.org/W2953327874","https://openalex.org/W2953873379","https://openalex.org/W2980383466","https://openalex.org/W2983273332","https://openalex.org/W3003858741","https://openalex.org/W3006355107","https://openalex.org/W3015727477","https://openalex.org/W3016219057","https://openalex.org/W3030235151","https://openalex.org/W3047322234","https://openalex.org/W3082667381","https://openalex.org/W3085858067","https://openalex.org/W3107705247","https://openalex.org/W3108630268","https://openalex.org/W3109812113","https://openalex.org/W3130293037","https://openalex.org/W3133595170","https://openalex.org/W3148835120","https://openalex.org/W3152848525","https://openalex.org/W3194784196","https://openalex.org/W3194922943","https://openalex.org/W4213293064","https://openalex.org/W4220995455","https://openalex.org/W4224235665","https://openalex.org/W4225265334","https://openalex.org/W4307407876","https://openalex.org/W4313294783","https://openalex.org/W4322769077","https://openalex.org/W4362458495","https://openalex.org/W4377220455","https://openalex.org/W4384131417","https://openalex.org/W4389974943","https://openalex.org/W4390100293","https://openalex.org/W4391378911","https://openalex.org/W4394721055","https://openalex.org/W4401011757","https://openalex.org/W4401994009","https://openalex.org/W4405022609","https://openalex.org/W4409886859","https://openalex.org/W4410041556","https://openalex.org/W4411339415","https://openalex.org/W4414446878","https://openalex.org/W7117451564","https://openalex.org/W7119481545","https://openalex.org/W7125233978","https://openalex.org/W7131354437","https://openalex.org/W7133871677","https://openalex.org/W7133928956","https://openalex.org/W7143416108","https://openalex.org/W7147184972","https://openalex.org/W7154576331","https://openalex.org/W7155701836"],"related_works":["https://openalex.org/W4411339415","https://openalex.org/W4389986208","https://openalex.org/W7204754786","https://openalex.org/W160362297","https://openalex.org/W4310464301","https://openalex.org/W7019192128","https://openalex.org/W2073387341","https://openalex.org/W3109812113","https://openalex.org/W2806601645","https://openalex.org/W2935896778"],"abstract_inverted_index":{"Abstract":[0],"Defects":[1],"in":[2,83,86,167,175,184,188,209],"lysosomal":[3],"lipid":[4],"handling":[5],"provoke":[6],"fatal":[7],"disorders":[8],"presenting":[9],"neurovisceral":[10],"symptoms":[11],"with":[12,154],"variable":[13],"onset":[14],"and":[15,31,67,70,96,136,145,173,203],"life":[16],"spans.":[17],"A":[18],"prime":[19],"example":[20],"is":[21,38,72],"Niemann-Pick":[22],"type":[23],"C":[24],"disease":[25],"(NPCD),":[26],"where":[27],"export":[28],"of":[29,43,94,103,117,127,148,152,170],"cholesterol":[30,47,54],"other":[32],"lipids":[33],"from":[34],"the":[35,113,128,176],"endosomal-lysosomal":[36],"system":[37],"impaired":[39],"due":[40],"to":[41,64],"variants":[42],"either":[44],"NPC":[45,52],"intracellular":[46,53],"transporter":[48,55],"1":[49],"(NPC1)":[50],"or":[51,133],"2":[56],"(NPC2).":[57],"Therapeutic":[58],"options":[59],"for":[60,76,201],"NPCD":[61,202],"are":[62],"limited":[63],"palliative":[65],"care":[66],"disease-modifying":[68],"drugs,":[69],"there":[71],"an":[73],"unmet":[74],"need":[75],"new":[77],"treatments.":[78],"Based":[79],"on":[80,141,159],"positive":[81],"effects":[82,139,158,181],"patient-derived":[84],"fibroblasts":[85],"vitro":[87],",":[88],"we":[89,122],"explored":[90],"here":[91],"how":[92],"inhibition":[93],"bromodomain":[95,194],"extra-terminal":[97],"domain":[98],"(BET)":[99],"proteins,":[100],"a":[101,108,164],"family":[102],"histone":[104],"acetylation":[105],"readers,":[106],"affects":[107],"well-established":[109],"mouse":[110],"model":[111],"bearing":[112],"frequent":[114],"I1061T":[115],"variant":[116],"NPC1.":[118],"To":[119],"this":[120],"end,":[121],"used":[123],"employed":[124],"intraperitoneal":[125],"administration":[126],"blood-brain-barrier":[129],"penetrating":[130],"drug":[131,138,199],"JQ1":[132,155],"vehicle":[134],"(dimethylsulfoxide),":[135],"gauged":[137],"based":[140],"weight,":[142,168],"biomarker":[143],"levels":[144],"quantitative":[146],"assessment":[147],"motor":[149],"coordination.":[150],"Treatment":[151],"mice":[153],"induced":[156],"beneficial":[157],"some":[160],"outcome":[161],"measures,":[162],"notably":[163],"transient":[165],"increase":[166],"reduction":[169],"liver":[171],"cholesterol,":[172],"improvements":[174],"footprint":[177],"test,":[178],"but":[179,186],"these":[180],"occurred":[182],"only":[183],"male":[185],"not":[187],"female":[189],"mice.":[190,210],"Our":[191],"results":[192],"indicate":[193],"proteins":[195],"as":[196],"potential":[197],"therapeutic":[198],"target":[200],"reveal":[204],"sex-dependent":[205],"BET":[206],"protein":[207],"signaling":[208]},"counts_by_year":[],"updated_date":"2026-11-09T13:22:03.289127","created_date":"2026-06-30T00:00:00"}