{"id":"https://openalex.org/W2014128251","doi":"https://doi.org/10.1038/mt.2015.6","title":"Adenoassociated Virus Serotype 9-Mediated Gene Therapy for X-Linked Adrenoleukodystrophy","display_name":"Adenoassociated Virus Serotype 9-Mediated Gene Therapy for X-Linked Adrenoleukodystrophy","publication_year":2015,"publication_date":"2015-01-16","ids":{"openalex":"https://openalex.org/W2014128251","doi":"https://doi.org/10.1038/mt.2015.6","mag":"2014128251","pmid":"https://pubmed.ncbi.nlm.nih.gov/25592337","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/4427888"},"language":"en","primary_location":{"id":"doi:10.1038/mt.2015.6","doi":"https://doi.org/10.1038/mt.2015.6","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2015.6","pdf_url":null,"source":{"id":"https://openalex.org/S51596929","display_name":"Molecular Therapy","issn_l":"1525-0016","issn":["1525-0016","1525-0024"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-3","ki-jl-2","medline","norway-1","poland-140","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":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://doi.org/10.1038/mt.2015.6","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5101624973","display_name":"Yi Gong","orcid":"https://orcid.org/0009-0005-5866-7873","observed_orcids":["https://orcid.org/0009-0005-5866-7873"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yi Gong","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020611876","display_name":"Dakai Mu","orcid":"https://orcid.org/0009-0005-7977-0941","observed_orcids":["https://orcid.org/0009-0005-7977-0941"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Dakai Mu","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5045740999","display_name":"Shilpa Prabhakar","orcid":"https://orcid.org/0000-0002-7999-2000","observed_orcids":["https://orcid.org/0000-0002-7999-2000"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Shilpa Prabhakar","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5059798104","display_name":"Ann B. Moser","orcid":"https://orcid.org/0000-0002-9147-1902","observed_orcids":["https://orcid.org/0000-0002-9147-1902"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Ann Moser","raw_affiliation_strings":["Peroxisome Disease Lab, Hugo W Moser Research Institute, Baltimore, Maryland, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Peroxisome Disease Lab, Hugo W Moser Research Institute, Baltimore, Maryland, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5029064436","display_name":"Patricia Leonor Musolino","orcid":"https://orcid.org/0000-0001-8724-5649","observed_orcids":["https://orcid.org/0000-0001-8724-5649"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Patricia Musolino","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100867341","display_name":"Jiaqian Ren","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"JiaQian Ren","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5059573074","display_name":"Xandra O. Breakefield","orcid":"https://orcid.org/0000-0001-6036-0399","observed_orcids":["https://orcid.org/0000-0001-6036-0399"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Xandra O Breakefield","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5064371575","display_name":"Casey A. Maguire","orcid":"https://orcid.org/0000-0001-8681-5179","observed_orcids":["https://orcid.org/0000-0001-8681-5179"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Casey A Maguire","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5036919823","display_name":"Florian Eichler","orcid":"https://orcid.org/0000-0001-6836-1089","observed_orcids":["https://orcid.org/0000-0001-6836-1089","https://orcid.org/0009-0001-2878-9758"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Florian S Eichler","raw_affiliation_strings":["Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA. Electronic address: feichler@partners.org","Department of Neurology, Massachusetts General Hospital , Boston, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA. Electronic address: feichler@partners.org","institution_ids":["https://openalex.org/I4210087915"]},{"raw_affiliation_string":"Department of Neurology, Massachusetts General Hospital , Boston, Massachusetts, USA","institution_ids":["https://openalex.org/I4210087915"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5036919823"],"corresponding_institution_ids":["https://openalex.org/I4210087915"],"apc_list":{"value":4500,"currency":"USD","value_usd":4500},"apc_paid":{"value":4500,"currency":"USD","value_usd":4500},"fwci":4.9628,"has_fulltext":false,"cited_by_count":74,"citation_normalized_percentile":{"value":0.96036098,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":95,"max":99},"biblio":{"volume":"23","issue":"5","first_page":"824","last_page":"834"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9883000254631042,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"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"}},"topics":[{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9883000254631042,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"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/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.005100000184029341,"subfield":{"id":"https://openalex.org/subfields/2737","display_name":"Physiology (medical)"},"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/T10725","display_name":"RNA Interference and Gene Delivery","score":0.0019000000320374966,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"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"}}],"keywords":[{"id":"https://openalex.org/keywords/x-linked-adrenoleukodystrophy","display_name":"X-linked adrenoleukodystrophy","score":0.9950000047683716},{"id":"https://openalex.org/keywords/aav9","display_name":"AAV9","score":0.8700000047683716},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.753000020980835},{"id":"https://openalex.org/keywords/abcd1-gene","display_name":"ABCD1 gene","score":0.9200000166893005},{"id":"https://openalex.org/keywords/very-long-chain-fatty-acids","display_name":"very long chain fatty acids","score":0.906000018119812},{"id":"https://openalex.org/keywords/intracerebroventricular-injection","display_name":"intracerebroventricular administration","score":0.906000018119812},{"id":"https://openalex.org/keywords/intravenous-injection","display_name":"intravenous injection","score":0.9739999771118164},{"id":"https://openalex.org/keywords/astrocytes","display_name":"astrocytes","score":0.7350000143051147},{"id":"https://openalex.org/keywords/microglia","display_name":"microglia","score":0.9399999976158142},{"id":"https://openalex.org/keywords/oligodendrocytes","display_name":"oligodendrocytes","score":0.9649999737739563},{"id":"https://openalex.org/keywords/adrenal-gland","display_name":"adrenal gland","score":0.878000020980835},{"id":"https://openalex.org/keywords/central-nervous-system-delivery","display_name":"central nervous system delivery","score":0.4869999885559082}],"concepts":[{"id":"https://openalex.org/C10389963","wikidata":"https://www.wikidata.org/wiki/Q848328","display_name":"Serotype","level":2,"score":0.780884861946106},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.7351082563400269},{"id":"https://openalex.org/C2777150068","wikidata":"https://www.wikidata.org/wiki/Q366964","display_name":"Adrenoleukodystrophy","level":4,"score":0.7105972766876221},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.6794043183326721},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.48627984523773193},{"id":"https://openalex.org/C2522874641","wikidata":"https://www.wikidata.org/wiki/Q808","display_name":"Virus","level":2,"score":0.47253039479255676},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.3786509335041046},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.3745880126953125},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.204437255859375},{"id":"https://openalex.org/C127078168","wikidata":"https://www.wikidata.org/wiki/Q138639","display_name":"Peroxisome","level":3,"score":0.0}],"mesh":[{"descriptor_ui":"D000074063","descriptor_name":"ATP Binding Cassette Transporter, Subfamily D, Member 1","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000094765","descriptor_name":"Glutathione Peroxidase GPX1","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000229","descriptor_name":"Dependovirus","qualifier_ui":"Q000145","qualifier_name":"classification","is_major_topic":false},{"descriptor_ui":"D000229","descriptor_name":"Dependovirus","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D000326","descriptor_name":"Adrenoleukodystrophy","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D000326","descriptor_name":"Adrenoleukodystrophy","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D001921","descriptor_name":"Brain","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D002478","descriptor_name":"Cells, Cultured","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004195","descriptor_name":"Disease Models, Animal","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005227","descriptor_name":"Fatty Acids","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D005347","descriptor_name":"Fibroblasts","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D005979","descriptor_name":"Glutathione Peroxidase","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009457","descriptor_name":"Neuroglia","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D014161","descriptor_name":"Transduction, Genetic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D015870","descriptor_name":"Gene Expression","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D017930","descriptor_name":"Genes, Reporter","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018345","descriptor_name":"Mice, Knockout","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018528","descriptor_name":"ATP-Binding Cassette Transporters","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D021381","descriptor_name":"Protein Transport","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D045744","descriptor_name":"Cell Line, Tumor","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":"D065288","descriptor_name":"Serogroup","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1038/mt.2015.6","doi":"https://doi.org/10.1038/mt.2015.6","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2015.6","pdf_url":null,"source":{"id":"https://openalex.org/S51596929","display_name":"Molecular Therapy","issn_l":"1525-0016","issn":["1525-0016","1525-0024"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-3","ki-jl-2","medline","norway-1","poland-140","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":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:4427888","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/4427888","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":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Mol Ther","raw_type":"Text"},{"id":"pmid:25592337","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/25592337","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 therapy : the journal of the American Society of Gene Therapy","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1038/mt.2015.6","doi":"https://doi.org/10.1038/mt.2015.6","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2015.6","pdf_url":null,"source":{"id":"https://openalex.org/S51596929","display_name":"Molecular Therapy","issn_l":"1525-0016","issn":["1525-0016","1525-0024"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-3","ki-jl-2","medline","norway-1","poland-140","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":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy","raw_type":"journal-article"},"sustainable_development_goals":[{"score":1.0,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1879129774","display_name":"A hybrid microvesicle/virus vector for targeted gene transfer to the brain","funder_award_id":"R21NS081374","funder_id":"https://openalex.org/F4320337359","funder_display_name":"National Institute of Neurological Disorders and Stroke"},{"id":"https://openalex.org/G2421180381","display_name":"Imaging the Pathophysiology of AMN in Mice and Humans","funder_award_id":"K08NS052550","funder_id":"https://openalex.org/F4320337359","funder_display_name":"National Institute of Neurological Disorders and Stroke"},{"id":"https://openalex.org/G4862919425","display_name":null,"funder_award_id":"NINDS P30NS045776","funder_id":"https://openalex.org/F4320337359","funder_display_name":"National Institute of Neurological Disorders and Stroke"},{"id":"https://openalex.org/G6789012777","display_name":"Interdepartmental Neuroscience Center","funder_award_id":"P30NS045776","funder_id":"https://openalex.org/F4320337359","funder_display_name":"National Institute of Neurological Disorders and Stroke"}],"funders":[{"id":"https://openalex.org/F4320306080","display_name":"Foundation for the National Institutes of Health","ror":"https://ror.org/00k86s890"},{"id":"https://openalex.org/F4320306596","display_name":"American Brain Tumor Association","ror":"https://ror.org/03w32n732"},{"id":"https://openalex.org/F4320337359","display_name":"National Institute of Neurological Disorders and Stroke","ror":"https://ror.org/01s5ya894"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":44,"referenced_works":["https://openalex.org/W1747533618","https://openalex.org/W1914200614","https://openalex.org/W1924236635","https://openalex.org/W1967242914","https://openalex.org/W1969962002","https://openalex.org/W1971793795","https://openalex.org/W1973343526","https://openalex.org/W1973418987","https://openalex.org/W1978221944","https://openalex.org/W1994710975","https://openalex.org/W1999567515","https://openalex.org/W2003200494","https://openalex.org/W2005093647","https://openalex.org/W2005872866","https://openalex.org/W2009016214","https://openalex.org/W2009235997","https://openalex.org/W2012428359","https://openalex.org/W2012894204","https://openalex.org/W2013503540","https://openalex.org/W2027414612","https://openalex.org/W2033005054","https://openalex.org/W2036030099","https://openalex.org/W2037167329","https://openalex.org/W2066299792","https://openalex.org/W2078815621","https://openalex.org/W2085106445","https://openalex.org/W2085854027","https://openalex.org/W2092757238","https://openalex.org/W2107786477","https://openalex.org/W2110746342","https://openalex.org/W2122304523","https://openalex.org/W2127566889","https://openalex.org/W2128033750","https://openalex.org/W2134199998","https://openalex.org/W2135907949","https://openalex.org/W2141570423","https://openalex.org/W2141759311","https://openalex.org/W2148470186","https://openalex.org/W2152163917","https://openalex.org/W2153279033","https://openalex.org/W2162290849","https://openalex.org/W2165825213","https://openalex.org/W2166739815","https://openalex.org/W2168163415"],"related_works":["https://openalex.org/W2946466199","https://openalex.org/W4416857331","https://openalex.org/W2561285251","https://openalex.org/W2898100827","https://openalex.org/W2104564418","https://openalex.org/W3208384289","https://openalex.org/W4407087930","https://openalex.org/W2599788389","https://openalex.org/W4403898721","https://openalex.org/W4388688001"],"abstract_inverted_index":{"X-linked":[0],"adrenoleukodystrophy":[1],"(X-ALD)":[2],"is":[3,174],"a":[4,17,96,195],"devastating":[5],"neurological":[6],"disorder":[7],"caused":[8],"by":[9,111],"mutations":[10],"in":[11,70,99,161,180,192],"the":[12,34,50,90,125,151,181],"ABCD1":[13,52,66,87,171],"gene":[14,53,67,172],"that":[15,169],"encodes":[16],"peroxisomal":[18],"ATP-binding":[19],"cassette":[20],"transporter":[21],"(ABCD1)":[22,54],"responsible":[23],"for":[24,36,47],"transport":[25],"of":[26,49,65,150,157,198],"CoA-activated":[27],"very":[28],"long-chain":[29],"fatty":[30],"acids":[31],"(VLCFA)":[32],"into":[33],"peroxisome":[35],"degradation.":[37],"We":[38,167],"used":[39],"recombinant":[40],"adenoassociated":[41],"virus":[42],"serotype":[43],"9":[44],"(rAAV9)":[45],"vector":[46],"delivery":[48,64],"human":[51,86],"to":[55,89,107,138,176],"mouse":[56,109,162,196],"central":[57],"nervous":[58,182],"system":[59,183],"(CNS).":[60],"In":[61,102],"vitro,":[62],"efficient":[63],"was":[68,105],"achieved":[69],"primary":[71],"mixed":[72],"brain":[73,163],"glial":[74],"cells":[75,141,179],"from":[76],"Abcd1-/-":[77,108],"mice":[78],"as":[79,81,187,189],"well":[80,188],"X-ALD":[82],"patient":[83],"fibroblasts.":[84],"Importantly,":[85,154],"localized":[88],"peroxisome,":[91],"and":[92,122,142,164,184,194],"AAV-ABCD1":[93],"transduction":[94,149],"showed":[95],"dose-dependent":[97],"effect":[98],"reducing":[100],"VLCFA.":[101],"vivo,":[103],"AAV9-ABCD1":[104,158],"delivered":[106,137],"CNS":[110],"either":[112],"stereotactic":[113],"intracerebroventricular":[114],"(ICV)":[115],"or":[116],"intravenous":[117],"(IV)":[118],"injections.":[119],"Astrocytes,":[120],"microglia":[121],"neurons":[123],"were":[124],"major":[126],"target":[127,178],"cell":[128],"types":[129],"following":[130],"ICV":[131],"injection,":[132],"while":[133],"IV":[134,144,155],"injection":[135,145,156],"also":[136,146],"microvascular":[139],"endothelial":[140],"oligodendrocytes.":[143],"yielded":[147],"high":[148],"adrenal":[152,185],"gland.":[153],"reduced":[159],"VLCFA":[160,191],"spinal":[165],"cord.":[166],"conclude":[168],"AAV9-mediated":[170],"transfer":[173],"able":[175],"reach":[177],"gland":[186],"reduce":[190],"culture":[193],"model":[197],"X-ALD.":[199]},"counts_by_year":[{"year":2026,"cited_by_count":4},{"year":2025,"cited_by_count":6},{"year":2024,"cited_by_count":14},{"year":2023,"cited_by_count":6},{"year":2022,"cited_by_count":5},{"year":2021,"cited_by_count":8},{"year":2020,"cited_by_count":3},{"year":2019,"cited_by_count":4},{"year":2018,"cited_by_count":4},{"year":2017,"cited_by_count":8},{"year":2016,"cited_by_count":9},{"year":2015,"cited_by_count":3}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2016-06-24T00:00:00"}