Get quick answers to your questions about the article from our AI researcher chatbot
{'id': 'https://openalex.org/W3203590990', 'doi': 'https://doi.org/10.1186/s12915-021-01133-x', 'title': 'A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1', 'display_name': 'A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1', 'publication_year': 2021, 'publication_date': '2021-10-01', 'ids': {'openalex': 'https://openalex.org/W3203590990', 'doi': 'https://doi.org/10.1186/s12915-021-01133-x', 'mag': '3203590990', 'pmid': 'https://pubmed.ncbi.nlm.nih.gov/34592985', 'pmcid': 'https://www.ncbi.nlm.nih.gov/pmc/articles/8485536'}, 'language': 'en', 'primary_location': {'is_oa': True, 'landing_page_url': 'https://doi.org/10.1186/s12915-021-01133-x', 'pdf_url': 'https://bmcbiol.biomedcentral.com/track/pdf/10.1186/s12915-021-01133-x', 'source': {'id': 'https://openalex.org/S25903957', 'display_name': 'BMC Biology', 'issn_l': '1741-7007', 'issn': ['1741-7007'], 'is_oa': True, 'is_in_doaj': True, 'is_core': True, 'host_organization': 'https://openalex.org/P4310320256', 'host_organization_name': 'BioMed Central', 'host_organization_lineage': ['https://openalex.org/P4310319965', 'https://openalex.org/P4310320256'], 'host_organization_lineage_names': ['Springer Nature', 'BioMed Central'], 'type': 'journal'}, 'license': 'cc-by', 'license_id': 'https://openalex.org/licenses/cc-by', 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, 'type': 'article', 'type_crossref': 'journal-article', 'indexed_in': ['crossref', 'doaj', 'pubmed'], 'open_access': {'is_oa': True, 'oa_status': 'gold', 'oa_url': 'https://bmcbiol.biomedcentral.com/track/pdf/10.1186/s12915-021-01133-x', 'any_repository_has_fulltext': True}, 'authorships': [{'author_position': 'first', 'author': {'id': 'https://openalex.org/A5021721257', 'display_name': 'Anika V. Prabhu', 'orcid': 'https://orcid.org/0000-0002-3809-1716'}, 'institutions': [{'id': 'https://openalex.org/I4210144228', 'display_name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development', 'ror': 'https://ror.org/04byxyr05', 'country_code': 'US', 'type': 'facility', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238', 'https://openalex.org/I4210144228']}, {'id': 'https://openalex.org/I1299303238', 'display_name': 'National Institutes of Health', 'ror': 'https://ror.org/01cwqze88', 'country_code': 'US', 'type': 'government', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Anika V. Prabhu', 'raw_affiliation_strings': ['Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA'], 'affiliations': [{'raw_affiliation_string': 'Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA', 'institution_ids': ['https://openalex.org/I4210144228', 'https://openalex.org/I1299303238']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5008590783', 'display_name': 'Insung Kang', 'orcid': 'https://orcid.org/0000-0003-1792-5002'}, 'institutions': [{'id': 'https://openalex.org/I4210144228', 'display_name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development', 'ror': 'https://ror.org/04byxyr05', 'country_code': 'US', 'type': 'facility', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238', 'https://openalex.org/I4210144228']}, {'id': 'https://openalex.org/I1299303238', 'display_name': 'National Institutes of Health', 'ror': 'https://ror.org/01cwqze88', 'country_code': 'US', 'type': 'government', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Insung Kang', 'raw_affiliation_strings': ['Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA'], 'affiliations': [{'raw_affiliation_string': 'Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA', 'institution_ids': ['https://openalex.org/I4210144228', 'https://openalex.org/I1299303238']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5045943797', 'display_name': 'Raffaella De Pace', 'orcid': 'https://orcid.org/0000-0002-8179-6869'}, 'institutions': [{'id': 'https://openalex.org/I4210144228', 'display_name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development', 'ror': 'https://ror.org/04byxyr05', 'country_code': 'US', 'type': 'facility', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238', 'https://openalex.org/I4210144228']}, {'id': 'https://openalex.org/I1299303238', 'display_name': 'National Institutes of Health', 'ror': 'https://ror.org/01cwqze88', 'country_code': 'US', 'type': 'government', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Raffaella De Pace', 'raw_affiliation_strings': ['Neurosciences and Cellular and Structural Biology Division, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, MD, 20892, USA'], 'affiliations': [{'raw_affiliation_string': 'Neurosciences and Cellular and Structural Biology Division, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, MD, 20892, USA', 'institution_ids': ['https://openalex.org/I4210144228', 'https://openalex.org/I1299303238']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5074217980', 'display_name': 'Christopher A. Wassif', 'orcid': 'https://orcid.org/0000-0002-2524-1420'}, 'institutions': [{'id': 'https://openalex.org/I4210144228', 'display_name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development', 'ror': 'https://ror.org/04byxyr05', 'country_code': 'US', 'type': 'facility', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238', 'https://openalex.org/I4210144228']}, {'id': 'https://openalex.org/I1299303238', 'display_name': 'National Institutes of Health', 'ror': 'https://ror.org/01cwqze88', 'country_code': 'US', 'type': 'government', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Christopher A. Wassif', 'raw_affiliation_strings': ['Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA'], 'affiliations': [{'raw_affiliation_string': 'Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA', 'institution_ids': ['https://openalex.org/I4210144228', 'https://openalex.org/I1299303238']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5021060288', 'display_name': 'Hideji Fujiwara', 'orcid': 'https://orcid.org/0000-0001-7694-142X'}, 'institutions': [{'id': 'https://openalex.org/I204465549', 'display_name': 'Washington University in St. Louis', 'ror': 'https://ror.org/01yc7t268', 'country_code': 'US', 'type': 'education', 'lineage': ['https://openalex.org/I204465549']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Hideji Fujiwara', 'raw_affiliation_strings': ['Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA'], 'affiliations': [{'raw_affiliation_string': 'Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA', 'institution_ids': ['https://openalex.org/I204465549']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5089158546', 'display_name': 'Pamela Kell', 'orcid': None}, 'institutions': [{'id': 'https://openalex.org/I204465549', 'display_name': 'Washington University in St. Louis', 'ror': 'https://ror.org/01yc7t268', 'country_code': 'US', 'type': 'education', 'lineage': ['https://openalex.org/I204465549']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Pamela Kell', 'raw_affiliation_strings': ['Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA'], 'affiliations': [{'raw_affiliation_string': 'Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA', 'institution_ids': ['https://openalex.org/I204465549']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5091851465', 'display_name': 'Xuntian Jiang', 'orcid': 'https://orcid.org/0000-0001-9048-7294'}, 'institutions': [{'id': 'https://openalex.org/I204465549', 'display_name': 'Washington University in St. Louis', 'ror': 'https://ror.org/01yc7t268', 'country_code': 'US', 'type': 'education', 'lineage': ['https://openalex.org/I204465549']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Xuntian Jiang', 'raw_affiliation_strings': ['Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA'], 'affiliations': [{'raw_affiliation_string': 'Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA', 'institution_ids': ['https://openalex.org/I204465549']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5060731094', 'display_name': 'Daniel S. Ory', 'orcid': 'https://orcid.org/0000-0002-9640-4901'}, 'institutions': [{'id': 'https://openalex.org/I204465549', 'display_name': 'Washington University in St. Louis', 'ror': 'https://ror.org/01yc7t268', 'country_code': 'US', 'type': 'education', 'lineage': ['https://openalex.org/I204465549']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Daniel S. Ory', 'raw_affiliation_strings': ['Diabetic Cardiovascular Disease Center, Washington University School of Medicine, St. Louis, MO, 63110, USA'], 'affiliations': [{'raw_affiliation_string': 'Diabetic Cardiovascular Disease Center, Washington University School of Medicine, St. Louis, MO, 63110, USA', 'institution_ids': ['https://openalex.org/I204465549']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5081989475', 'display_name': 'Juan S. Bonifacino', 'orcid': 'https://orcid.org/0000-0002-5673-6370'}, 'institutions': [{'id': 'https://openalex.org/I4210144228', 'display_name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development', 'ror': 'https://ror.org/04byxyr05', 'country_code': 'US', 'type': 'facility', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238', 'https://openalex.org/I4210144228']}, {'id': 'https://openalex.org/I1299303238', 'display_name': 'National Institutes of Health', 'ror': 'https://ror.org/01cwqze88', 'country_code': 'US', 'type': 'government', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Juan S. Bonifacino', 'raw_affiliation_strings': ['Neurosciences and Cellular and Structural Biology Division, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, MD, 20892, USA'], 'affiliations': [{'raw_affiliation_string': 'Neurosciences and Cellular and Structural Biology Division, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, MD, 20892, USA', 'institution_ids': ['https://openalex.org/I4210144228', 'https://openalex.org/I1299303238']}]}, {'author_position': 'middle', 'author': {'id': 'https://openalex.org/A5027663167', 'display_name': 'Michael E. Ward', 'orcid': 'https://orcid.org/0000-0002-5296-8051'}, 'institutions': [{'id': 'https://openalex.org/I4210110767', 'display_name': 'National Institute of Neurological Disorders and Stroke', 'ror': 'https://ror.org/01s5ya894', 'country_code': 'US', 'type': 'facility', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238', 'https://openalex.org/I4210110767']}, {'id': 'https://openalex.org/I1299303238', 'display_name': 'National Institutes of Health', 'ror': 'https://ror.org/01cwqze88', 'country_code': 'US', 'type': 'government', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Michael E. Ward', 'raw_affiliation_strings': ['National Institute of Neurological Disorders and Stroke, National Institutes of Health, DHHS, Bethesda, MD, 20892, USA'], 'affiliations': [{'raw_affiliation_string': 'National Institute of Neurological Disorders and Stroke, National Institutes of Health, DHHS, Bethesda, MD, 20892, USA', 'institution_ids': ['https://openalex.org/I4210110767', 'https://openalex.org/I1299303238']}]}, {'author_position': 'last', 'author': {'id': 'https://openalex.org/A5043346952', 'display_name': 'Forbes D. Porter', 'orcid': 'https://orcid.org/0000-0001-9397-0046'}, 'institutions': [{'id': 'https://openalex.org/I4210144228', 'display_name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development', 'ror': 'https://ror.org/04byxyr05', 'country_code': 'US', 'type': 'facility', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238', 'https://openalex.org/I4210144228']}, {'id': 'https://openalex.org/I1299303238', 'display_name': 'National Institutes of Health', 'ror': 'https://ror.org/01cwqze88', 'country_code': 'US', 'type': 'government', 'lineage': ['https://openalex.org/I1299022934', 'https://openalex.org/I1299303238']}], 'countries': ['US'], 'is_corresponding': False, 'raw_author_name': 'Forbes D. Porter', 'raw_affiliation_strings': ['Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA'], 'affiliations': [{'raw_affiliation_string': 'Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, 10CRC, Rm. 5-2571, 10 Center Dr, Bethesda, MD, USA', 'institution_ids': ['https://openalex.org/I4210144228', 'https://openalex.org/I1299303238']}]}], 'institution_assertions': [], 'countries_distinct_count': 1, 'institutions_distinct_count': 4, 'corresponding_author_ids': [], 'corresponding_institution_ids': [], 'apc_list': {'value': 2290, 'currency': 'GBP', 'value_usd': 2808, 'provenance': 'doaj'}, 'apc_paid': {'value': 2290, 'currency': 'GBP', 'value_usd': 2808, 'provenance': 'doaj'}, 'fwci': 1.741, 'has_fulltext': True, 'fulltext_origin': 'pdf', 'cited_by_count': 12, 'citation_normalized_percentile': {'value': 0.754601, 'is_in_top_1_percent': False, 'is_in_top_10_percent': False}, 'cited_by_percentile_year': {'min': 89, 'max': 90}, 'biblio': {'volume': '19', 'issue': '1', 'first_page': None, 'last_page': None}, 'is_retracted': False, 'is_paratext': False, 'primary_topic': {'id': 'https://openalex.org/T10945', 'display_name': 'Lysosomal Storage Disorders Research', 'score': 0.9999, 'subfield': {'id': 'https://openalex.org/subfields/2737', 'display_name': 'Physiology'}, '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': 0.9999, 'subfield': {'id': 'https://openalex.org/subfields/2737', 'display_name': 'Physiology'}, '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': 0.9567, 'subfield': {'id': 'https://openalex.org/subfields/2745', 'display_name': 'Rheumatology'}, '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/T11184', 'display_name': 'Neonatal and fetal brain pathology', 'score': 0.9552, 'subfield': {'id': 'https://openalex.org/subfields/2735', 'display_name': 'Pediatrics, Perinatology and Child Health'}, '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/human-disease', 'display_name': 'Human disease', 'score': 0.44280827}], 'concepts': [{'id': 'https://openalex.org/C86803240', 'wikidata': 'https://www.wikidata.org/wiki/Q420', 'display_name': 'Biology', 'level': 0, 'score': 0.9405085}, {'id': 'https://openalex.org/C2779134260', 'wikidata': 'https://www.wikidata.org/wiki/Q12136', 'display_name': 'Disease', 'level': 2, 'score': 0.66349286}, {'id': 'https://openalex.org/C2780635549', 'wikidata': 'https://www.wikidata.org/wiki/Q2067267', 'display_name': 'Niemann–Pick disease, type C', 'level': 3, 'score': 0.52645785}, {'id': 'https://openalex.org/C169760540', 'wikidata': 'https://www.wikidata.org/wiki/Q207011', 'display_name': 'Neuroscience', 'level': 1, 'score': 0.47228914}, {'id': 'https://openalex.org/C2988867146', 'wikidata': 'https://www.wikidata.org/wiki/Q12136', 'display_name': 'Human disease', 'level': 3, 'score': 0.44280827}, {'id': 'https://openalex.org/C2777299769', 'wikidata': 'https://www.wikidata.org/wiki/Q3707858', 'display_name': 'Type (biology)', 'level': 2, 'score': 0.4369085}, {'id': 'https://openalex.org/C70721500', 'wikidata': 'https://www.wikidata.org/wiki/Q177005', 'display_name': 'Computational biology', 'level': 1, 'score': 0.3236577}, {'id': 'https://openalex.org/C54355233', 'wikidata': 'https://www.wikidata.org/wiki/Q7162', 'display_name': 'Genetics', 'level': 1, 'score': 0.27686977}, {'id': 'https://openalex.org/C104317684', 'wikidata': 'https://www.wikidata.org/wiki/Q7187', 'display_name': 'Gene', 'level': 2, 'score': 0.16255715}, {'id': 'https://openalex.org/C126322002', 'wikidata': 'https://www.wikidata.org/wiki/Q11180', 'display_name': 'Internal medicine', 'level': 1, 'score': 0.15548778}, {'id': 'https://openalex.org/C55493867', 'wikidata': 'https://www.wikidata.org/wiki/Q7094', 'display_name': 'Biochemistry', 'level': 1, 'score': 0.11969721}, {'id': 'https://openalex.org/C18903297', 'wikidata': 'https://www.wikidata.org/wiki/Q7150', 'display_name': 'Ecology', 'level': 1, 'score': 0.08041018}, {'id': 'https://openalex.org/C2778163477', 'wikidata': 'https://www.wikidata.org/wiki/Q43656', 'display_name': 'Cholesterol', 'level': 2, 'score': 0.07168576}, {'id': 'https://openalex.org/C71924100', 'wikidata': 'https://www.wikidata.org/wiki/Q11190', 'display_name': 'Medicine', 'level': 0, 'score': 0.0}], 'mesh': [{'descriptor_ui': 'D057026', 'descriptor_name': 'Induced Pluripotent Stem Cells', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': True}, {'descriptor_ui': 'D052556', 'descriptor_name': 'Niemann-Pick Disease, Type C', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': True}, {'descriptor_ui': 'D002784', 'descriptor_name': 'Cholesterol', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D006801', 'descriptor_name': 'Humans', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D009474', 'descriptor_name': 'Neurons', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}, {'descriptor_ui': 'D052556', 'descriptor_name': 'Niemann-Pick Disease, Type C', 'qualifier_ui': 'Q000235', 'qualifier_name': 'genetics', 'is_major_topic': False}, {'descriptor_ui': 'D004364', 'descriptor_name': 'Pharmaceutical Preparations', 'qualifier_ui': '', 'qualifier_name': None, 'is_major_topic': False}], 'locations_count': 4, 'locations': [{'is_oa': True, 'landing_page_url': 'https://doi.org/10.1186/s12915-021-01133-x', 'pdf_url': 'https://bmcbiol.biomedcentral.com/track/pdf/10.1186/s12915-021-01133-x', 'source': {'id': 'https://openalex.org/S25903957', 'display_name': 'BMC Biology', 'issn_l': '1741-7007', 'issn': ['1741-7007'], 'is_oa': True, 'is_in_doaj': True, 'is_core': True, 'host_organization': 'https://openalex.org/P4310320256', 'host_organization_name': 'BioMed Central', 'host_organization_lineage': ['https://openalex.org/P4310319965', 'https://openalex.org/P4310320256'], 'host_organization_lineage_names': ['Springer Nature', 'BioMed Central'], 'type': 'journal'}, 'license': 'cc-by', 'license_id': 'https://openalex.org/licenses/cc-by', 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, {'is_oa': False, 'landing_page_url': 'https://doaj.org/article/fbf727df91264aa1910bff5cd9a48708', 'pdf_url': None, 'source': {'id': 'https://openalex.org/S4306401280', 'display_name': 'DOAJ (DOAJ: Directory of Open Access Journals)', 'issn_l': None, 'issn': None, 'is_oa': True, 'is_in_doaj': False, 'is_core': False, 'host_organization': None, 'host_organization_name': None, 'host_organization_lineage': [], 'host_organization_lineage_names': [], 'type': 'repository'}, 'license': None, 'license_id': None, 'version': None, 'is_accepted': False, 'is_published': False}, {'is_oa': True, 'landing_page_url': 'https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8485536', 'pdf_url': None, 'source': {'id': 'https://openalex.org/S2764455111', 'display_name': 'PubMed Central', 'issn_l': None, 'issn': None, 'is_oa': True, 'is_in_doaj': False, 'is_core': False, 'host_organization': 'https://openalex.org/I1299303238', 'host_organization_name': 'National Institutes of Health', 'host_organization_lineage': ['https://openalex.org/I1299303238'], 'host_organization_lineage_names': ['National Institutes of Health'], 'type': 'repository'}, 'license': None, 'license_id': None, 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, {'is_oa': False, 'landing_page_url': 'https://pubmed.ncbi.nlm.nih.gov/34592985', 'pdf_url': None, 'source': {'id': 'https://openalex.org/S4306525036', 'display_name': 'PubMed', 'issn_l': None, 'issn': None, 'is_oa': False, 'is_in_doaj': False, 'is_core': False, 'host_organization': 'https://openalex.org/I1299303238', 'host_organization_name': 'National Institutes of Health', 'host_organization_lineage': ['https://openalex.org/I1299303238'], 'host_organization_lineage_names': ['National Institutes of Health'], 'type': 'repository'}, 'license': None, 'license_id': None, 'version': None, 'is_accepted': False, 'is_published': False}], 'best_oa_location': {'is_oa': True, 'landing_page_url': 'https://doi.org/10.1186/s12915-021-01133-x', 'pdf_url': 'https://bmcbiol.biomedcentral.com/track/pdf/10.1186/s12915-021-01133-x', 'source': {'id': 'https://openalex.org/S25903957', 'display_name': 'BMC Biology', 'issn_l': '1741-7007', 'issn': ['1741-7007'], 'is_oa': True, 'is_in_doaj': True, 'is_core': True, 'host_organization': 'https://openalex.org/P4310320256', 'host_organization_name': 'BioMed Central', 'host_organization_lineage': ['https://openalex.org/P4310319965', 'https://openalex.org/P4310320256'], 'host_organization_lineage_names': ['Springer Nature', 'BioMed Central'], 'type': 'journal'}, 'license': 'cc-by', 'license_id': 'https://openalex.org/licenses/cc-by', 'version': 'publishedVersion', 'is_accepted': True, 'is_published': True}, 'sustainable_development_goals': [{'id': 'https://metadata.un.org/sdg/3', 'display_name': 'Good health and well-being', 'score': 0.64}], 'grants': [{'funder': 'https://openalex.org/F4320306794', 'funder_display_name': 'Ara Parseghian Medical Research Foundation', 'award_id': None}, {'funder': 'https://openalex.org/F4320332161', 'funder_display_name': 'National Institutes of Health', 'award_id': None}], 'datasets': ['https://openalex.org/W4394528730', 'https://openalex.org/W4394477986'], 'versions': [], 'referenced_works_count': 39, 'referenced_works': ['https://openalex.org/W146706964', 'https://openalex.org/W1623462303', 'https://openalex.org/W1690219722', 'https://openalex.org/W1971365671', 'https://openalex.org/W1973260880', 'https://openalex.org/W2006098335', 'https://openalex.org/W2011555635', 'https://openalex.org/W2011892782', 'https://openalex.org/W2031211461', 'https://openalex.org/W2040407461', 'https://openalex.org/W2042422091', 'https://openalex.org/W2045652399', 'https://openalex.org/W2049312906', 'https://openalex.org/W2059999994', 'https://openalex.org/W2064978465', 'https://openalex.org/W2065408118', 'https://openalex.org/W2068262899', 'https://openalex.org/W2093934810', 'https://openalex.org/W2097259163', 'https://openalex.org/W2106070783', 'https://openalex.org/W2119814716', 'https://openalex.org/W2147612731', 'https://openalex.org/W2367049629', 'https://openalex.org/W2416068580', 'https://openalex.org/W2499571652', 'https://openalex.org/W2521862911', 'https://openalex.org/W2554496886', 'https://openalex.org/W2560057938', 'https://openalex.org/W2571918371', 'https://openalex.org/W2744065025', 'https://openalex.org/W2760524820', 'https://openalex.org/W2790775254', 'https://openalex.org/W2803819386', 'https://openalex.org/W2806809913', 'https://openalex.org/W2885234628', 'https://openalex.org/W2921423796', 'https://openalex.org/W2924919479', 'https://openalex.org/W2954000601', 'https://openalex.org/W2967789674'], 'related_works': ['https://openalex.org/W4393222579', 'https://openalex.org/W3204004755', 'https://openalex.org/W3203590990', 'https://openalex.org/W3135344376', 'https://openalex.org/W3084201033', 'https://openalex.org/W2344443137', 'https://openalex.org/W2343122611', 'https://openalex.org/W2121343109', 'https://openalex.org/W2102522934', 'https://openalex.org/W2068262899'], 'abstract_inverted_index': {'Abstract': [0], 'Background': [1], 'Niemann-Pick': [2, 135], 'disease,': [3, 99, 136], 'type': [4, 137], 'C': [5], '(NPC)': [6], 'is': [7], 'a': [8, 30, 127, 143, 237, 249, 283], 'childhood-onset,': [9], 'lethal,': [10], 'neurodegenerative': [11, 95], 'disorder': [12], 'caused': [13], 'by': [14, 26, 233], 'autosomal': [15], 'recessive': [16], 'mutations': [17, 43], 'in': [18, 44, 58, 66, 105, 188, 243, 248, 264], 'the': [19, 59, 74, 94, 101, 111, 124, 166, 257, 287, 293], 'genes': [20], 'NPC1': [21, 46, 152, 163, 169, 191, 199, 244, 274, 288], 'or': [22, 47], 'NPC2': [23, 48], 'and': [24, 42, 53, 63, 78, 117, 165, 212, 217, 226, 247], 'characterized': [25], 'impaired': [27, 218], 'cholesterol': [28, 37, 57], 'homeostasis,': [29], 'lipid': [31], 'essential': [32], 'for': [33, 80, 103, 286], 'cellular': [34, 76, 112, 198], 'function.': [35], 'Cellular': [36], 'levels': [38], 'are': [39], 'tightly': [40], 'regulated,': [41], 'either': [45], 'lead': [49], 'to': [50, 72, 178, 268, 291, 297], 'deficient': [51], 'transport': [52], 'accumulation': [54, 209], 'of': [55, 97, 126, 134, 160, 206, 210, 259], 'unesterified': [56, 207], 'late': [60], 'endosome/lysosome': [61], 'compartment,': [62], 'progressive': [64], 'neurodegeneration': [65], 'affected': [67], 'individuals.': [68], 'Previous': [69], 'cell-based': [70], 'studies': [71], 'understand': [73], 'NPC': [75, 98], 'pathophysiology': [77], 'screen': [79], 'therapeutic': [81, 271], 'agents': [82], 'have': [83], 'mainly': [84], 'used': [85, 177, 187], 'patient': [86], 'fibroblasts.': [87], 'However,': [88], 'these': [89], 'do': [90], 'not': [91], 'allow': [92], 'modeling': [93], 'aspect': [96], 'highlighting': [100], 'need': [102], 'an': [104], 'vitro': [106], 'system': [107], 'that': [108, 184, 239], 'permits': [109], 'understanding': [110], 'mechanisms': [113, 295], 'underlying': [114], 'neuronal': [115, 132, 298], 'loss': [116], 'identifying': [118], 'appropriate': [119], 'therapies.': [120], 'This': [121], 'study': [122], 'reports': [123], 'development': [125], 'novel': [128], 'human': [129], 'iPSC-derived,': [130], 'inducible': [131], 'model': [133, 159], 'C1': [138], '(NPC1).': [139], 'Results': [140], 'We': [141], 'generated': [142], 'null': [144], 'i3Neuron': [145], '(inducible': [146], '×': [147, 149], 'integrated': [148], 'isogenic)': [150], '(': [151], '−/−': [153, 164, 192, 275], 'i': [154, 171, 193, 276], '3': [155, 172, 194, 277], 'Neuron)': [156], 'iPSC-derived': [157], 'neuron': [158], 'NPC1.': [161], 'The': [162, 273], 'corresponding': [167], 'isogenic': [168], '+/+': [170], 'Neuron': [173, 278], 'cell': [174, 262], 'lines': [175], 'were': [176], 'efficiently': [179], 'generate': [180], 'homogenous,': [181], 'synchronized': [182], 'neurons': [183], 'can': [185, 230], 'be': [186, 231, 282], 'high-throughput': [189, 265], 'screens.': [190], 'Neurons': [195], 'recapitulate': [196], 'cardinal': [197], 'pathological': [200, 294], 'features': [201], 'including': [202], 'perinuclear': [203], 'endolysosomal': [204], 'storage': [205], 'cholesterol,': [208], 'GM2': [211], 'GM3': [213], 'gangliosides,': [214], 'mitochondrial': [215, 224], 'dysfunction,': [216, 225], 'axonal': [219, 227], 'lysosomal': [220], 'transport.': [221], 'Cholesterol': [222], 'storage,': [223], 'trafficking': [228], 'defects': [229], 'ameliorated': [232], 'treatment': [234], 'with': [235], '2-hydroxypropyl-β-cyclodextrin,': [236], 'drug': [238], 'has': [240], 'shown': [241], 'efficacy': [242], 'preclinical': [245], 'models': [246], 'phase': [250], '1/2a': [251], 'trial.': [252], 'Conclusion': [253], 'Our': [254], 'data': [255], 'demonstrate': [256], 'utility': [258], 'this': [260], 'new': [261], 'line': [263, 279], 'drug/chemical': [266], 'screens': [267], 'identify': [269], 'potential': [270], 'agents.': [272], 'will': [280], 'also': [281], 'valuable': [284], 'tool': [285], 'research': [289], 'community': [290], 'explore': [292], 'contributing': [296], 'degeneration.': [299], 'Graphical': [300], 'abstract': [301]}, 'cited_by_api_url': 'https://api.openalex.org/works?filter=cites:W3203590990', 'counts_by_year': [{'year': 2024, 'cited_by_count': 6}, {'year': 2023, 'cited_by_count': 2}, {'year': 2022, 'cited_by_count': 4}], 'updated_date': '2025-01-06T18:58:34.871048', 'created_date': '2021-10-11'}