{"id":"https://openalex.org/W2000488765","doi":"https://doi.org/10.1172/jci1773","title":"Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.","display_name":"Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.","publication_year":1998,"publication_date":"1998-04-01","ids":{"openalex":"https://openalex.org/W2000488765","doi":"https://doi.org/10.1172/jci1773","mag":"2000488765","pmid":"https://pubmed.ncbi.nlm.nih.gov/9525982"},"language":"en","primary_location":{"id":"doi:10.1172/jci1773","doi":"https://doi.org/10.1172/jci1773","is_oa":false,"landing_page_url":"https://doi.org/10.1172/jci1773","pdf_url":null,"source":{"id":"https://openalex.org/S114430552","display_name":"Journal of Clinical Investigation","issn_l":"0021-9738","issn":["0021-9738","1558-8238"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320279","host_organization_name":"American Society for Clinical Investigation","host_organization_lineage":["https://openalex.org/P4310320279"],"host_organization_lineage_names":["American Society for Clinical Investigation"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Journal of Clinical Investigation","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/508717","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5110227314","display_name":"Lynn H. O\u2019Connor","orcid":null,"observed_orcids":[]},"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":"L H O'Connor","raw_affiliation_strings":["Department of Psychiatry, Washington University School of Medicine, St. Louis, Missouri 63110, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Psychiatry, Washington University School of Medicine, St. Louis, Missouri 63110, USA","institution_ids":["https://openalex.org/I204465549"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5109911048","display_name":"Lawrence C. Erway","orcid":null,"observed_orcids":[]},"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":"L C Erway","raw_affiliation_strings":["Washington Uni-versity School of Medicine, Department of Internal Medicine, 660 South Euclid Avenue, Box 8007, St. Louis, MO 63110"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Washington Uni-versity School of Medicine, Department of Internal Medicine, 660 South Euclid Avenue, Box 8007, St. Louis, MO 63110","institution_ids":["https://openalex.org/I204465549"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5082333244","display_name":"Carole A. Vogler","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"C A Vogler","raw_affiliation_strings":["Department of Pathology, and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pathology, and","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113555411","display_name":"William S. Sly","orcid":null,"observed_orcids":[]},"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":"W S Sly","raw_affiliation_strings":["Washington Uni-versity School of Medicine, Department of Internal Medicine, 660 South Euclid Avenue, Box 8007, St. Louis, MO 63110"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Washington Uni-versity School of Medicine, Department of Internal Medicine, 660 South Euclid Avenue, Box 8007, St. Louis, MO 63110","institution_ids":["https://openalex.org/I204465549"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5005302667","display_name":"Andrew Nicholes","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"A Nicholes","raw_affiliation_strings":["Department of Internal Medicine, and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Internal Medicine, and","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020402727","display_name":"Jeffrey H. Grubb","orcid":null,"observed_orcids":[]},"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":"J Grubb","raw_affiliation_strings":["Washington Uni-versity School of Medicine, Department of Internal Medicine, 660 South Euclid Avenue, Box 8007, St. Louis, MO 63110"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Washington Uni-versity School of Medicine, Department of Internal Medicine, 660 South Euclid Avenue, Box 8007, St. Louis, MO 63110","institution_ids":["https://openalex.org/I204465549"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111644087","display_name":"Sandra W. Holmberg","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I47838141","display_name":"Saint Louis University","ror":"https://ror.org/01p7jjy08","country_code":"US","type":"education","lineage":["https://openalex.org/I47838141"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"S W Holmberg","raw_affiliation_strings":["Department of Psychiatry,","The Edward A. Doisy Department of Biochemistry and Molecular Biology, St. Louis University School of Medicine, St. Louis, Missouri 63104"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Psychiatry,","institution_ids":[]},{"raw_affiliation_string":"The Edward A. Doisy Department of Biochemistry and Molecular Biology, St. Louis University School of Medicine, St. Louis, Missouri 63104","institution_ids":["https://openalex.org/I47838141"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5068778015","display_name":"Beth K. Levy","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"B Levy","raw_affiliation_strings":["Department of Pathology, and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pathology, and","institution_ids":[]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5049345275","display_name":"Mark S. Sands","orcid":"https://orcid.org/0000-0002-5559-0832","observed_orcids":["https://orcid.org/0000-0002-5559-0832"]},"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"]},{"id":"https://openalex.org/I63135867","display_name":"University of Cincinnati","ror":"https://ror.org/01e3m7079","country_code":"US","type":"education","lineage":["https://openalex.org/I63135867"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"M S Sands","raw_affiliation_strings":["Department of Genetics, Washington University School of Medicine, St. Louis, Missouri 63110; \u0288 Department of Biological Sciences,","Department of Internal Medicine, and","University of Cincinnati, Cincinnati, Ohio 45221;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Genetics, Washington University School of Medicine, St. Louis, Missouri 63110; \u0288 Department of Biological Sciences,","institution_ids":["https://openalex.org/I204465549"]},{"raw_affiliation_string":"Department of Internal Medicine, and","institution_ids":[]},{"raw_affiliation_string":"University of Cincinnati, Cincinnati, Ohio 45221;","institution_ids":["https://openalex.org/I63135867"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5049345275"],"corresponding_institution_ids":["https://openalex.org/I204465549","https://openalex.org/I63135867"],"apc_list":{"value":5200,"currency":"USD","value_usd":5200},"apc_paid":null,"fwci":9.4321,"has_fulltext":true,"cited_by_count":113,"citation_normalized_percentile":{"value":0.98387097,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":88,"max":97},"biblio":{"volume":"101","issue":"7","first_page":"1394","last_page":"1400"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":1.0,"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":1.0,"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":9.999999747378752e-05,"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/T14415","display_name":"Biomedical Research and Pathophysiology","score":0.0,"subfield":{"id":"https://openalex.org/subfields/2734","display_name":"Pathology and Forensic Medicine"},"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/mucopolysaccharidosis-type-vii","display_name":"mucopolysaccharidosis type VII","score":0.996999979019165},{"id":"https://openalex.org/keywords/sly-syndrome","display_name":"Sly syndrome","score":0.9380000233650208},{"id":"https://openalex.org/keywords/enzyme-replacement-therapy","display_name":"enzyme replacement therapy","score":0.9539999961853027},{"id":"https://openalex.org/keywords/lysosomal-storage-disorders","display_name":"lysosomal storage disease","score":0.8840000033378601},{"id":"https://openalex.org/keywords/hearing-loss","display_name":"hearing loss","score":0.6899999976158142},{"id":"https://openalex.org/keywords/auditory-function","display_name":"auditory function","score":0.6909999847412109},{"id":"https://openalex.org/keywords/learning-and-memory","display_name":"learning and memory","score":0.8270000219345093},{"id":"https://openalex.org/keywords/behavioral-deficits","display_name":"behavioral deficits","score":0.7419999837875366},{"id":"https://openalex.org/keywords/early-treatment-initiation","display_name":"early treatment initiation","score":0.5230000019073486},{"id":"https://openalex.org/keywords/mouse-model","display_name":"mouse model","score":0.5960000157356262}],"concepts":[{"id":"https://openalex.org/C2779969927","wikidata":"https://www.wikidata.org/wiki/Q916766","display_name":"Enzyme replacement therapy","level":3,"score":0.9388153553009033},{"id":"https://openalex.org/C2779416428","wikidata":"https://www.wikidata.org/wiki/Q1479681","display_name":"Mucopolysaccharidosis","level":2,"score":0.7753586769104004},{"id":"https://openalex.org/C2778264360","wikidata":"https://www.wikidata.org/wiki/Q675010","display_name":"Lysosomal storage disease","level":3,"score":0.720399022102356},{"id":"https://openalex.org/C2910727661","wikidata":"https://www.wikidata.org/wiki/Q25379699","display_name":"Mucopolysaccharidosis I","level":4,"score":0.6481693387031555},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.5499089956283569},{"id":"https://openalex.org/C44752575","wikidata":"https://www.wikidata.org/wiki/Q1417562","display_name":"Morris water navigation task","level":3,"score":0.5049131512641907},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.4715566635131836},{"id":"https://openalex.org/C2780493683","wikidata":"https://www.wikidata.org/wiki/Q16035842","display_name":"Hearing loss","level":2,"score":0.46428006887435913},{"id":"https://openalex.org/C2911091166","wikidata":"https://www.wikidata.org/wiki/Q106419912","display_name":"Transplantation","level":2,"score":0.45979827642440796},{"id":"https://openalex.org/C181199279","wikidata":"https://www.wikidata.org/wiki/Q8047","display_name":"Enzyme","level":2,"score":0.44810691475868225},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.39627280831336975},{"id":"https://openalex.org/C134018914","wikidata":"https://www.wikidata.org/wiki/Q162606","display_name":"Endocrinology","level":1,"score":0.3643295466899872},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.3532094359397888},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.20293763279914856},{"id":"https://openalex.org/C548259974","wikidata":"https://www.wikidata.org/wiki/Q569965","display_name":"Audiology","level":1,"score":0.20149007439613342},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.1491900384426117},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.12036880850791931},{"id":"https://openalex.org/C148762608","wikidata":"https://www.wikidata.org/wiki/Q3748185","display_name":"Hippocampal formation","level":2,"score":0.0}],"mesh":[{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D001522","descriptor_name":"Behavior, Animal","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D001522","descriptor_name":"Behavior, Animal","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D001522","descriptor_name":"Behavior, Animal","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000201","qualifier_name":"enzymology","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000201","qualifier_name":"enzymology","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000201","qualifier_name":"enzymology","is_major_topic":false},{"descriptor_ui":"D004423","descriptor_name":"Ear","qualifier_ui":"Q000033","qualifier_name":"anatomy & histology","is_major_topic":false},{"descriptor_ui":"D004423","descriptor_name":"Ear","qualifier_ui":"Q000033","qualifier_name":"anatomy & histology","is_major_topic":false},{"descriptor_ui":"D004423","descriptor_name":"Ear","qualifier_ui":"Q000033","qualifier_name":"anatomy & histology","is_major_topic":false},{"descriptor_ui":"D005966","descriptor_name":"Glucuronidase","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":false},{"descriptor_ui":"D005966","descriptor_name":"Glucuronidase","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":false},{"descriptor_ui":"D005966","descriptor_name":"Glucuronidase","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":false},{"descriptor_ui":"D006309","descriptor_name":"Hearing","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D006309","descriptor_name":"Hearing","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D006309","descriptor_name":"Hearing","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D007275","descriptor_name":"Injections, Intravenous","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007275","descriptor_name":"Injections, Intravenous","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007275","descriptor_name":"Injections, Intravenous","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007858","descriptor_name":"Learning","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D007858","descriptor_name":"Learning","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D007858","descriptor_name":"Learning","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D008247","descriptor_name":"Lysosomes","qualifier_ui":"Q000648","qualifier_name":"ultrastructure","is_major_topic":false},{"descriptor_ui":"D008247","descriptor_name":"Lysosomes","qualifier_ui":"Q000648","qualifier_name":"ultrastructure","is_major_topic":false},{"descriptor_ui":"D008247","descriptor_name":"Lysosomes","qualifier_ui":"Q000648","qualifier_name":"ultrastructure","is_major_topic":false},{"descriptor_ui":"D008817","descriptor_name":"Mice, Mutant Strains","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008817","descriptor_name":"Mice, Mutant Strains","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008817","descriptor_name":"Mice, Mutant Strains","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D010641","descriptor_name":"Phenotype","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D010641","descriptor_name":"Phenotype","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D010641","descriptor_name":"Phenotype","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D013997","descriptor_name":"Time Factors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D013997","descriptor_name":"Time Factors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D013997","descriptor_name":"Time Factors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016538","descriptor_name":"Mucopolysaccharidosis VII","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D016538","descriptor_name":"Mucopolysaccharidosis VII","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D016538","descriptor_name":"Mucopolysaccharidosis VII","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D016538","descriptor_name":"Mucopolysaccharidosis VII","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D016538","descriptor_name":"Mucopolysaccharidosis VII","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D016538","descriptor_name":"Mucopolysaccharidosis VII","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1172/jci1773","doi":"https://doi.org/10.1172/jci1773","is_oa":false,"landing_page_url":"https://doi.org/10.1172/jci1773","pdf_url":null,"source":{"id":"https://openalex.org/S114430552","display_name":"Journal of Clinical Investigation","issn_l":"0021-9738","issn":["0021-9738","1558-8238"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320279","host_organization_name":"American Society for Clinical Investigation","host_organization_lineage":["https://openalex.org/P4310320279"],"host_organization_lineage_names":["American Society for Clinical Investigation"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Journal of Clinical Investigation","raw_type":"journal-article"},{"id":"pmid:9525982","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/9525982","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"The Journal of clinical investigation","raw_type":"Journal Article"},{"id":"pmh:oai:pubmedcentral.nih.gov:508717","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/508717","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"J Clin Invest","raw_type":"Text"}],"best_oa_location":{"id":"pmh:oai:pubmedcentral.nih.gov:508717","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/508717","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"J Clin Invest","raw_type":"Text"},"sustainable_development_goals":[{"score":0.9789000153541565,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"sustainable_development_goals_aurora":[{"score":0.7300000190734863,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G3046823453","display_name":null,"funder_award_id":"DK50158","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"},{"id":"https://openalex.org/G3334678270","display_name":"Characterization and Treatment of Murine MPS VII","funder_award_id":"5r01dk041082-15","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G3658087230","display_name":null,"funder_award_id":"DK-41082","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"},{"id":"https://openalex.org/G4186911987","display_name":null,"funder_award_id":"GM34182","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"},{"id":"https://openalex.org/G8531198242","display_name":"GENE THERAPY STRATEGIES FOR MUCOPOLYSACCHARIDOSIS VII","funder_award_id":"1r55dk050158-01a1","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"}],"funders":[{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320337354","display_name":"National Institute of General Medical Sciences","ror":"https://ror.org/04q48ey07"},{"id":"https://openalex.org/F4320337357","display_name":"National Institute of Diabetes and Digestive and Kidney Diseases","ror":"https://ror.org/00adh9b73"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":46,"referenced_works":["https://openalex.org/W1481493576","https://openalex.org/W1482169420","https://openalex.org/W1507750508","https://openalex.org/W1541926252","https://openalex.org/W1559407499","https://openalex.org/W1596137101","https://openalex.org/W1599431022","https://openalex.org/W1968067472","https://openalex.org/W1970030078","https://openalex.org/W1973986115","https://openalex.org/W1978903497","https://openalex.org/W1996543665","https://openalex.org/W2006038562","https://openalex.org/W2020872680","https://openalex.org/W2025319847","https://openalex.org/W2027832018","https://openalex.org/W2028533348","https://openalex.org/W2035161172","https://openalex.org/W2035814440","https://openalex.org/W2038155620","https://openalex.org/W2042557135","https://openalex.org/W2045112729","https://openalex.org/W2050142708","https://openalex.org/W2052347675","https://openalex.org/W2058581959","https://openalex.org/W2062963785","https://openalex.org/W2063049455","https://openalex.org/W2066146740","https://openalex.org/W2066913836","https://openalex.org/W2070981327","https://openalex.org/W2071940392","https://openalex.org/W2073304593","https://openalex.org/W2076633759","https://openalex.org/W2078976766","https://openalex.org/W2088789807","https://openalex.org/W2095158220","https://openalex.org/W2140984295","https://openalex.org/W2169473035","https://openalex.org/W2256622923","https://openalex.org/W2257523867","https://openalex.org/W2322128806","https://openalex.org/W2412476834","https://openalex.org/W2414492394","https://openalex.org/W2415790124","https://openalex.org/W3145894777","https://openalex.org/W4254413115"],"related_works":["https://openalex.org/W2125045778","https://openalex.org/W2294727991","https://openalex.org/W2327110361","https://openalex.org/W2029085773","https://openalex.org/W2048686056","https://openalex.org/W2321043350","https://openalex.org/W2002593000","https://openalex.org/W2028706623","https://openalex.org/W2000488765","https://openalex.org/W4238863489"],"abstract_inverted_index":{"Mucopolysaccharidosis":[0],"type":[1],"VII":[2,46,79,103,107,121,140,168],"(MPS":[3],"VII;":[4],"Sly":[5],"syndrome)":[6],"is":[7,174],"one":[8],"of":[9,12,31,40,44,65,75,126,137,156],"a":[10,41],"group":[11],"lysosomal":[13],"storage":[14,28],"diseases":[15],"that":[16,87,154],"share":[17],"many":[18],"clinical":[19],"features,":[20],"including":[21],"mental":[22],"retardation":[23],"and":[24,34,97,114,145,160,188],"hearing":[25,99,161],"loss.":[26],"Lysosomal":[27],"in":[29,77,101,123,166,177],"neurons":[30],"the":[32,35,72,94,127,134,157,186],"brain":[33],"associated":[36],"behavioral":[37,95],"abnormalities":[38],"characteristic":[39],"murine":[42],"model":[43],"MPS":[45,78,102,106,120,139,167],"have":[47],"not":[48],"been":[49],"shown":[50],"to":[51,111,185],"be":[52,164],"corrected":[53],"by":[54],"either":[55],"bone":[56],"marrow":[57],"transplantation":[58],"or":[59],"gene":[60],"therapy.":[61,195],"However,":[62],"intravenous":[63],"injections":[64],"recombinant":[66],"beta-glucuronidase":[67],"initiated":[68,90,175],"at":[69,91],"birth":[70,92],"reduce":[71],"pathological":[73],"evidence":[74,86],"disease":[76],"mice.":[80,104,150],"In":[81,132],"this":[82],"study":[83],"we":[84],"present":[85],"enzyme":[88,171,193],"replacement":[89,172,194],"improved":[93],"performance":[96],"reduced":[98],"loss":[100],"Enzyme-treated":[105],"mice":[108,113,122,141,169],"performed":[109],"similarly":[110],"normal":[112,149],"significantly":[115],"better":[116],"than":[117],"mock-":[118],"treated":[119,138],"every":[124],"phase":[125],"Morris":[128],"Water":[129],"Maze":[130],"test.":[131],"addition,":[133],"auditory":[135],"function":[136],"was":[142,146],"dramatically":[143],"improved,":[144],"indistinguishable":[147],"from":[148],"These":[151,179],"data":[152,180],"indicate":[153],"some":[155],"learning,":[158],"memory,":[159],"deficits":[162],"can":[163],"prevented":[165],"if":[170],"therapy":[173],"early":[176],"life.":[178],"also":[181],"provide":[182],"functional":[183],"correlates":[184],"biochemical":[187],"histopathological":[189],"improvements":[190],"observed":[191],"after":[192]},"counts_by_year":[{"year":2025,"cited_by_count":1},{"year":2022,"cited_by_count":1},{"year":2020,"cited_by_count":3},{"year":2019,"cited_by_count":1},{"year":2018,"cited_by_count":2},{"year":2016,"cited_by_count":3},{"year":2015,"cited_by_count":2},{"year":2014,"cited_by_count":3},{"year":2013,"cited_by_count":3},{"year":2012,"cited_by_count":3}],"updated_date":"2026-10-16T09:59:22.235173","created_date":"2025-10-10T00:00:00"}
