{"id":"https://openalex.org/W2052298217","doi":"https://doi.org/10.1016/j.ymgme.2014.09.004","title":"Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis","display_name":"Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis","publication_year":2014,"publication_date":"2014-09-16","ids":{"openalex":"https://openalex.org/W2052298217","doi":"https://doi.org/10.1016/j.ymgme.2014.09.004","mag":"2052298217","pmid":"https://pubmed.ncbi.nlm.nih.gov/25257657"},"language":"en","primary_location":{"id":"doi:10.1016/j.ymgme.2014.09.004","doi":"https://doi.org/10.1016/j.ymgme.2014.09.004","is_oa":false,"landing_page_url":"https://doi.org/10.1016/j.ymgme.2014.09.004","pdf_url":null,"source":{"id":"https://openalex.org/S42433034","display_name":"Molecular Genetics and Metabolism","issn_l":"1096-7192","issn":["1096-7192","1096-7206"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Genetics and Metabolism","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":false,"oa_status":"closed","oa_url":null,"any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5005739369","display_name":"Brian R. Vuillemenot","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Brian R. Vuillemenot","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: bvuillemenot@bmrn.com"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: bvuillemenot@bmrn.com","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Derek Kennedy","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Derek Kennedy","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5070668450","display_name":"Jonathan D. Cooper","orcid":"https://orcid.org/0000-0003-1339-4750","observed_orcids":["https://orcid.org/0000-0003-1339-4750"]},"institutions":[{"id":"https://openalex.org/I183935753","display_name":"King's College London","ror":"https://ror.org/0220mzb33","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I183935753"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Jonathan D. Cooper","raw_affiliation_strings":["King's College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"King's College London, London, UK","institution_ids":["https://openalex.org/I183935753"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5103453508","display_name":"Andrew M.S. Wong","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I183935753","display_name":"King's College London","ror":"https://ror.org/0220mzb33","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I183935753"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Andrew M.S. Wong","raw_affiliation_strings":["King's College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"King's College London, London, UK","institution_ids":["https://openalex.org/I183935753"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110862099","display_name":"Sarmi Sri","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I183935753","display_name":"King's College London","ror":"https://ror.org/0220mzb33","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I183935753"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Sarmi Sri","raw_affiliation_strings":["King's College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"King's College London, London, UK","institution_ids":["https://openalex.org/I183935753"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5003339442","display_name":"Thom Doeleman","orcid":"https://orcid.org/0000-0003-4568-9510","observed_orcids":["https://orcid.org/0000-0003-4568-9510"]},"institutions":[{"id":"https://openalex.org/I183935753","display_name":"King's College London","ror":"https://ror.org/0220mzb33","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I183935753"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Thom Doeleman","raw_affiliation_strings":["King's College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"King's College London, London, UK","institution_ids":["https://openalex.org/I183935753"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5074790517","display_name":"Martin L. Katz","orcid":"https://orcid.org/0000-0002-2582-9187","observed_orcids":["https://orcid.org/0000-0002-2582-9187"]},"institutions":[{"id":"https://openalex.org/I76835614","display_name":"University of Missouri","ror":"https://ror.org/02ymw8z06","country_code":"US","type":"education","lineage":["https://openalex.org/I76835614"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Martin L. Katz","raw_affiliation_strings":["University of Missouri, Columbia, MO, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Missouri, Columbia, MO, USA","institution_ids":["https://openalex.org/I76835614"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5002088032","display_name":"Joan R. Coates","orcid":"https://orcid.org/0000-0002-9267-5528","observed_orcids":["https://orcid.org/0000-0002-9267-5528"]},"institutions":[{"id":"https://openalex.org/I76835614","display_name":"University of Missouri","ror":"https://ror.org/02ymw8z06","country_code":"US","type":"education","lineage":["https://openalex.org/I76835614"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Joan R. Coates","raw_affiliation_strings":["University of Missouri, Columbia, MO, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Missouri, Columbia, MO, USA","institution_ids":["https://openalex.org/I76835614"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113691877","display_name":"Gayle C. Johnson","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I76835614","display_name":"University of Missouri","ror":"https://ror.org/02ymw8z06","country_code":"US","type":"education","lineage":["https://openalex.org/I76835614"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Gayle C. Johnson","raw_affiliation_strings":["University of Missouri, Columbia, MO, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Missouri, Columbia, MO, USA","institution_ids":["https://openalex.org/I76835614"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5015516561","display_name":"Randall P. Reed","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Randall P. Reed","raw_affiliation_strings":["Northern Biomedical Research, Muskegon, MI, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Northern Biomedical Research, Muskegon, MI, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5057979971","display_name":"Eric L. Adams","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Eric L. Adams","raw_affiliation_strings":["Northern Biomedical Research, Muskegon, MI, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Northern Biomedical Research, Muskegon, MI, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5070412458","display_name":"Mark T. Butt","orcid":"https://orcid.org/0000-0001-8613-199X","observed_orcids":["https://orcid.org/0000-0001-8613-199X"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Mark T. Butt","raw_affiliation_strings":["Tox Path Specialists, Frederick, MD, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Tox Path Specialists, Frederick, MD, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5032921714","display_name":"Donald G. Musson","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Donald G. Musson","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110532943","display_name":"Joshua Henshaw","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Joshua Henshaw","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5075473838","display_name":"Steve Keve","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Steve Keve","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5066506744","display_name":"Rhea Cahayag","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Rhea Cahayag","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5075917653","display_name":"Laurie Tsuruda","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Laurie S. Tsuruda","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA","institution_ids":["https://openalex.org/I91786662"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5112736126","display_name":"Charles A. O\u2019neill","orcid":"https://orcid.org/0000-0003-2586-6874","observed_orcids":["https://orcid.org/0000-0003-2586-6874"]},"institutions":[{"id":"https://openalex.org/I91786662","display_name":"BioMarin (United States)","ror":"https://ror.org/03fmvqd28","country_code":"US","type":"company","lineage":["https://openalex.org/I91786662"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Charles A. O'Neill","raw_affiliation_strings":["BioMarin Pharmaceutical Inc., Novato, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"BioMarin Pharmaceutical Inc., Novato, CA, USA","institution_ids":["https://openalex.org/I91786662"]}]}],"institutions":[],"countries_distinct_count":2,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5005739369"],"corresponding_institution_ids":["https://openalex.org/I91786662"],"apc_list":{"value":3680,"currency":"USD","value_usd":3680},"apc_paid":null,"fwci":4.4676,"has_fulltext":false,"cited_by_count":60,"citation_normalized_percentile":{"value":0.94769127,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":89,"max":99},"biblio":{"volume":"114","issue":"2","first_page":"281","last_page":"293"},"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/T11027","display_name":"Metabolism and Genetic Disorders","score":0.0,"subfield":{"id":"https://openalex.org/subfields/1308","display_name":"Clinical 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/T12604","display_name":"Glycogen Storage Diseases and Myoclonus","score":0.0,"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"}}],"keywords":[{"id":"https://openalex.org/keywords/batten-disease","display_name":"Batten disease","score":0.9020000100135803},{"id":"https://openalex.org/keywords/tripeptidyl-peptidase-1","display_name":"tripeptidyl peptidase 1","score":0.968999981880188},{"id":"https://openalex.org/keywords/intracerebroventricular-injection","display_name":"intracerebroventricular administration","score":0.8100000023841858},{"id":"https://openalex.org/keywords/cerebrospinal-fluid-pharmacokinetics","display_name":"cerebrospinal fluid pharmacokinetics","score":0.8560000061988831},{"id":"https://openalex.org/keywords/anti-drug-antibodies","display_name":"anti-drug antibodies","score":0.7360000014305115},{"id":"https://openalex.org/keywords/neuroinflammation","display_name":"neuroinflammation","score":0.8450000286102295},{"id":"https://openalex.org/keywords/neurodegeneration","display_name":"neurodegeneration","score":0.6510000228881836},{"id":"https://openalex.org/keywords/lysosomal-storage-disorders","display_name":"lysosomal storage disease","score":0.6330000162124634}],"concepts":[{"id":"https://openalex.org/C2778230176","wikidata":"https://www.wikidata.org/wiki/Q1753778","display_name":"Batten disease","level":3,"score":0.9411218762397766},{"id":"https://openalex.org/C2778650512","wikidata":"https://www.wikidata.org/wiki/Q4358039","display_name":"Neuronal ceroid lipofuscinosis","level":3,"score":0.8027238249778748},{"id":"https://openalex.org/C2779651940","wikidata":"https://www.wikidata.org/wiki/Q54196","display_name":"Cerebrospinal fluid","level":2,"score":0.6942688822746277},{"id":"https://openalex.org/C2776925932","wikidata":"https://www.wikidata.org/wiki/Q1755122","display_name":"Neurodegeneration","level":3,"score":0.682177722454071},{"id":"https://openalex.org/C2779969927","wikidata":"https://www.wikidata.org/wiki/Q916766","display_name":"Enzyme replacement therapy","level":3,"score":0.5939648747444153},{"id":"https://openalex.org/C112705442","wikidata":"https://www.wikidata.org/wiki/Q323936","display_name":"Pharmacokinetics","level":2,"score":0.5522124171257019},{"id":"https://openalex.org/C529278444","wikidata":"https://www.wikidata.org/wiki/Q47273","display_name":"Central nervous system","level":2,"score":0.49685052037239075},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.46904000639915466},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.4528906047344208},{"id":"https://openalex.org/C134018914","wikidata":"https://www.wikidata.org/wiki/Q162606","display_name":"Endocrinology","level":1,"score":0.43328163027763367},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.3888854384422302},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.3102113604545593}],"mesh":[{"descriptor_ui":"D000091346","descriptor_name":"Tripeptidyl-Peptidase 1","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000091346","descriptor_name":"Tripeptidyl-Peptidase 1","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000091346","descriptor_name":"Tripeptidyl-Peptidase 1","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D000626","descriptor_name":"Aminopeptidases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","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":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000906","descriptor_name":"Antibodies","qualifier_ui":"Q000097","qualifier_name":"blood","is_major_topic":false},{"descriptor_ui":"D000906","descriptor_name":"Antibodies","qualifier_ui":"Q000097","qualifier_name":"blood","is_major_topic":false},{"descriptor_ui":"D000906","descriptor_name":"Antibodies","qualifier_ui":"Q000097","qualifier_name":"blood","is_major_topic":false},{"descriptor_ui":"D000906","descriptor_name":"Antibodies","qualifier_ui":"Q000134","qualifier_name":"cerebrospinal fluid","is_major_topic":false},{"descriptor_ui":"D000906","descriptor_name":"Antibodies","qualifier_ui":"Q000134","qualifier_name":"cerebrospinal fluid","is_major_topic":false},{"descriptor_ui":"D000906","descriptor_name":"Antibodies","qualifier_ui":"Q000134","qualifier_name":"cerebrospinal fluid","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000648","qualifier_name":"ultrastructure","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000648","qualifier_name":"ultrastructure","is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000648","qualifier_name":"ultrastructure","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D004152","descriptor_name":"Dipeptidyl-Peptidases and Tripeptidyl-Peptidases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D004285","descriptor_name":"Dogs","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004285","descriptor_name":"Dogs","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004285","descriptor_name":"Dogs","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004353","descriptor_name":"Drug Evaluation, Preclinical","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004353","descriptor_name":"Drug Evaluation, Preclinical","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004353","descriptor_name":"Drug Evaluation, Preclinical","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005838","descriptor_name":"Genotype","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005838","descriptor_name":"Genotype","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005838","descriptor_name":"Genotype","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009472","descriptor_name":"Neuronal Ceroid-Lipofuscinoses","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":false},{"descriptor_ui":"D009472","descriptor_name":"Neuronal Ceroid-Lipofuscinoses","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":false},{"descriptor_ui":"D009472","descriptor_name":"Neuronal Ceroid-Lipofuscinoses","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":false},{"descriptor_ui":"D009472","descriptor_name":"Neuronal Ceroid-Lipofuscinoses","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D009472","descriptor_name":"Neuronal Ceroid-Lipofuscinoses","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D009472","descriptor_name":"Neuronal Ceroid-Lipofuscinoses","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D011994","descriptor_name":"Recombinant Proteins","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D018450","descriptor_name":"Disease Progression","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018450","descriptor_name":"Disease Progression","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018450","descriptor_name":"Disease Progression","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D056947","descriptor_name":"Enzyme Replacement Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D056947","descriptor_name":"Enzyme Replacement Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D056947","descriptor_name":"Enzyme Replacement Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D057057","descriptor_name":"Serine Proteases","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D057967","descriptor_name":"Infusions, Intraventricular","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D057967","descriptor_name":"Infusions, Intraventricular","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D057967","descriptor_name":"Infusions, Intraventricular","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1016/j.ymgme.2014.09.004","doi":"https://doi.org/10.1016/j.ymgme.2014.09.004","is_oa":false,"landing_page_url":"https://doi.org/10.1016/j.ymgme.2014.09.004","pdf_url":null,"source":{"id":"https://openalex.org/S42433034","display_name":"Molecular Genetics and Metabolism","issn_l":"1096-7192","issn":["1096-7192","1096-7206"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Genetics and Metabolism","raw_type":"journal-article"},{"id":"pmid:25257657","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/25257657","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":"Molecular genetics and metabolism","raw_type":"Journal Article"},{"id":"pmh:oai:kclpure.kcl.ac.uk:publications/a99c2693-210f-4ebf-b382-a0b2fd14f3d1","doi":null,"is_oa":false,"landing_page_url":"https://kclpure.kcl.ac.uk/portal/en/publications/a99c2693-210f-4ebf-b382-a0b2fd14f3d1","pdf_url":null,"source":{"id":"https://openalex.org/S4306400216","display_name":"Research Portal (King's College London)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I183935753","host_organization_name":"King's College London","host_organization_lineage":["https://openalex.org/I183935753"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Vuillemenot, B R, Kennedy, D, Cooper, J D, Wong, A M S, Sri, S, Doeleman, T, Katz, M L, Coates, J R, Johnson, G C, Reed, R P, Adams, E L, Butt, M T, Musson, D G, Henshaw, J, Keve, S, Cahayag, R, Tsuruda, L S & O'Neill, C A 2015, 'Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis', MOLECULAR GENETICS AND METABOLISM, vol. 114, no. 2, pp. 281-293. https://doi.org/10.1016/j.ymgme.2014.09.004","raw_type":"info:eu-repo/semantics/article"}],"best_oa_location":null,"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","score":0.9922999739646912,"display_name":"Good health and well-being"}],"sustainable_development_goals_aurora":[{"id":"https://metadata.un.org/sdg/3","score":0.8299999833106995,"display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[{"id":"https://openalex.org/F4320310557","display_name":"BioMarin Pharmaceutical","ror":"https://ror.org/03fmvqd28"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":39,"referenced_works":["https://openalex.org/W113901592","https://openalex.org/W1502830907","https://openalex.org/W1547781044","https://openalex.org/W1602997642","https://openalex.org/W1867061005","https://openalex.org/W1916006974","https://openalex.org/W1933952120","https://openalex.org/W1967259894","https://openalex.org/W1967567480","https://openalex.org/W1975017859","https://openalex.org/W1985349046","https://openalex.org/W1991467400","https://openalex.org/W2010395737","https://openalex.org/W2020278806","https://openalex.org/W2026420637","https://openalex.org/W2027013896","https://openalex.org/W2027407881","https://openalex.org/W2036844023","https://openalex.org/W2046218561","https://openalex.org/W2060441085","https://openalex.org/W2065711632","https://openalex.org/W2068588733","https://openalex.org/W2071543365","https://openalex.org/W2073128198","https://openalex.org/W2077824751","https://openalex.org/W2078557934","https://openalex.org/W2079877218","https://openalex.org/W2083479642","https://openalex.org/W2100636864","https://openalex.org/W2107128771","https://openalex.org/W2110746342","https://openalex.org/W2114016428","https://openalex.org/W2138308725","https://openalex.org/W2156144629","https://openalex.org/W2165937627","https://openalex.org/W2466382613","https://openalex.org/W2777106254","https://openalex.org/W4211166988","https://openalex.org/W6811408596"],"related_works":["https://openalex.org/W2317527551","https://openalex.org/W2166453547","https://openalex.org/W2126741328","https://openalex.org/W2082887407","https://openalex.org/W2124523908","https://openalex.org/W3016158054","https://openalex.org/W82223775","https://openalex.org/W2163557498","https://openalex.org/W1974168805","https://openalex.org/W2953171110"],"abstract_inverted_index":null,"counts_by_year":[{"year":2026,"cited_by_count":2},{"year":2025,"cited_by_count":3},{"year":2024,"cited_by_count":5},{"year":2023,"cited_by_count":2},{"year":2022,"cited_by_count":7},{"year":2021,"cited_by_count":2},{"year":2020,"cited_by_count":6},{"year":2019,"cited_by_count":8},{"year":2018,"cited_by_count":2},{"year":2017,"cited_by_count":8},{"year":2016,"cited_by_count":7},{"year":2015,"cited_by_count":7},{"year":2014,"cited_by_count":1}],"updated_date":"2026-10-16T09:59:22.235173","created_date":"2025-10-10T00:00:00"}