{"id":"https://openalex.org/W1970617299","doi":"https://doi.org/10.1016/j.ymthe.2004.02.024","title":"Retroviral-mediated gene transfer restores IL-12 and IL-23 signaling pathways in T cells from IL-12 receptor \u03b21-deficient patients","display_name":"Retroviral-mediated gene transfer restores IL-12 and IL-23 signaling pathways in T cells from IL-12 receptor \u03b21-deficient patients","publication_year":2004,"publication_date":"2004-04-28","ids":{"openalex":"https://openalex.org/W1970617299","doi":"https://doi.org/10.1016/j.ymthe.2004.02.024","mag":"1970617299","pmid":"https://pubmed.ncbi.nlm.nih.gov/15194056"},"language":"en","primary_location":{"id":"doi:10.1016/j.ymthe.2004.02.024","doi":"https://doi.org/10.1016/j.ymthe.2004.02.024","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.ymthe.2004.02.024","pdf_url":"http://www.cell.com/article/S1525001604001005/pdf","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":"http://www.cell.com/article/S1525001604001005/pdf","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5021653930","display_name":"Marita Bosticardo","orcid":"https://orcid.org/0000-0002-1771-4523","observed_orcids":["https://orcid.org/0000-0002-1771-4523"]},"institutions":[{"id":"https://openalex.org/I1299303238","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88","country_code":"US","type":"government","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238"]},{"id":"https://openalex.org/I4210090236","display_name":"National Human Genome Research Institute","ror":"https://ror.org/00baak391","country_code":"US","type":"facility","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238","https://openalex.org/I4210090236"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Marita Bosticardo","raw_affiliation_strings":["Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, Building 49, Room 3A20, MSC 4442, Bethesda, MD 20892-4442, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, Building 49, Room 3A20, MSC 4442, Bethesda, MD 20892-4442, USA","institution_ids":["https://openalex.org/I1299303238","https://openalex.org/I4210090236"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5002880319","display_name":"Iren Witte","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1299303238","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88","country_code":"US","type":"government","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238"]},{"id":"https://openalex.org/I4210090236","display_name":"National Human Genome Research Institute","ror":"https://ror.org/00baak391","country_code":"US","type":"facility","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238","https://openalex.org/I4210090236"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Iren Witte","raw_affiliation_strings":["Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, Building 49, Room 3A20, MSC 4442, Bethesda, MD 20892-4442, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, Building 49, Room 3A20, MSC 4442, Bethesda, MD 20892-4442, USA","institution_ids":["https://openalex.org/I1299303238","https://openalex.org/I4210090236"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5017403173","display_name":"Claire Fieschi","orcid":"https://orcid.org/0000-0003-2136-6178","observed_orcids":["https://orcid.org/0000-0003-2136-6178"]},"institutions":[{"id":"https://openalex.org/I154526488","display_name":"Inserm","ror":"https://ror.org/02vjkv261","country_code":"FR","type":"government","lineage":["https://openalex.org/I154526488"]},{"id":"https://openalex.org/I4210162046","display_name":"Human Genetic of Infectious Diseases","ror":"https://ror.org/04z4fen17","country_code":"FR","type":"facility","lineage":["https://openalex.org/I154526488","https://openalex.org/I4210155509","https://openalex.org/I4210162046"]},{"id":"https://openalex.org/I4387156284","display_name":"Universit\u00e9 Paris Descartes","ror":"https://ror.org/0220k9r37","country_code":"FR","type":"education","lineage":["https://openalex.org/I204730241","https://openalex.org/I4387156284"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Claire Fieschi","raw_affiliation_strings":["Laboratoire de G\u00e9n\u00e9tique Humaine des Maladies Infectieuses, Universit\u00e9 Ren\u00e9 Descartes\u2013INSERM U550, Facult\u00e9 de M\u00e9decine Necker, 75015 Paris, France, EU"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Laboratoire de G\u00e9n\u00e9tique Humaine des Maladies Infectieuses, Universit\u00e9 Ren\u00e9 Descartes\u2013INSERM U550, Facult\u00e9 de M\u00e9decine Necker, 75015 Paris, France, EU","institution_ids":["https://openalex.org/I154526488","https://openalex.org/I4210162046","https://openalex.org/I4387156284"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5040743234","display_name":"Francesco Novelli","orcid":"https://orcid.org/0000-0002-6259-5666","observed_orcids":["https://orcid.org/0000-0002-6259-5666"]},"institutions":[{"id":"https://openalex.org/I4210097265","display_name":"Azienda Ospedaliero Universitaria San Giovanni Battista","ror":"https://ror.org/00nrtez23","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I4210097265"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"Francesco Novelli","raw_affiliation_strings":["Center for Experimental Research and Clinical Studies, San Giovanni Battista Hospital, Turin 10126, Italy, EU"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Experimental Research and Clinical Studies, San Giovanni Battista Hospital, Turin 10126, Italy, EU","institution_ids":["https://openalex.org/I4210097265"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5047928635","display_name":"Jean\u2010Laurent Casanova","orcid":"https://orcid.org/0000-0002-7782-4169","observed_orcids":["https://orcid.org/0000-0002-7782-4169"]},"institutions":[{"id":"https://openalex.org/I154526488","display_name":"Inserm","ror":"https://ror.org/02vjkv261","country_code":"FR","type":"government","lineage":["https://openalex.org/I154526488"]},{"id":"https://openalex.org/I4210162046","display_name":"Human Genetic of Infectious Diseases","ror":"https://ror.org/04z4fen17","country_code":"FR","type":"facility","lineage":["https://openalex.org/I154526488","https://openalex.org/I4210155509","https://openalex.org/I4210162046"]},{"id":"https://openalex.org/I4387156284","display_name":"Universit\u00e9 Paris Descartes","ror":"https://ror.org/0220k9r37","country_code":"FR","type":"education","lineage":["https://openalex.org/I204730241","https://openalex.org/I4387156284"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"Jean-Laurent Casanova","raw_affiliation_strings":["Laboratoire de G\u00e9n\u00e9tique Humaine des Maladies Infectieuses, Universit\u00e9 Ren\u00e9 Descartes\u2013INSERM U550, Facult\u00e9 de M\u00e9decine Necker, 75015 Paris, France, EU"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Laboratoire de G\u00e9n\u00e9tique Humaine des Maladies Infectieuses, Universit\u00e9 Ren\u00e9 Descartes\u2013INSERM U550, Facult\u00e9 de M\u00e9decine Necker, 75015 Paris, France, EU","institution_ids":["https://openalex.org/I154526488","https://openalex.org/I4210162046","https://openalex.org/I4387156284"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5062385004","display_name":"Fabio Candotti","orcid":"https://orcid.org/0000-0001-6399-6042","observed_orcids":["https://orcid.org/0000-0001-6399-6042"]},"institutions":[{"id":"https://openalex.org/I1299303238","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88","country_code":"US","type":"government","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238"]},{"id":"https://openalex.org/I4210090236","display_name":"National Human Genome Research Institute","ror":"https://ror.org/00baak391","country_code":"US","type":"facility","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238","https://openalex.org/I4210090236"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Fabio Candotti","raw_affiliation_strings":["Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, Building 49, Room 3A20, MSC 4442, Bethesda, MD 20892-4442, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, Building 49, Room 3A20, MSC 4442, Bethesda, MD 20892-4442, USA","institution_ids":["https://openalex.org/I1299303238","https://openalex.org/I4210090236"]}]}],"institutions":[],"countries_distinct_count":3,"institutions_distinct_count":6,"corresponding_author_ids":["https://openalex.org/A5062385004"],"corresponding_institution_ids":["https://openalex.org/I1299303238","https://openalex.org/I4210090236"],"apc_list":{"value":4500,"currency":"USD","value_usd":4500},"apc_paid":{"value":4500,"currency":"USD","value_usd":4500},"fwci":0.8893,"has_fulltext":false,"cited_by_count":10,"citation_normalized_percentile":{"value":0.71984984,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":89,"max":94},"biblio":{"volume":"9","issue":"6","first_page":"895","last_page":"901"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9731000065803528,"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":0.9731000065803528,"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/T11725","display_name":"Immunodeficiency and Autoimmune Disorders","score":0.009600000455975533,"subfield":{"id":"https://openalex.org/subfields/2403","display_name":"Immunology"},"field":{"id":"https://openalex.org/fields/24","display_name":"Immunology and Microbiology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10580","display_name":"Immunotherapy and Immune Responses","score":0.003100000089034438,"subfield":{"id":"https://openalex.org/subfields/2403","display_name":"Immunology"},"field":{"id":"https://openalex.org/fields/24","display_name":"Immunology and Microbiology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/retroviral-gene-transfer","display_name":"retroviral gene transfer","score":0.8880000114440918},{"id":"https://openalex.org/keywords/il-12-signaling","display_name":"IL-12 signaling","score":0.9120000004768372},{"id":"https://openalex.org/keywords/il-23-signaling","display_name":"IL-23 signaling","score":0.9990000128746033},{"id":"https://openalex.org/keywords/t-cells","display_name":"T cells","score":0.7879999876022339},{"id":"https://openalex.org/keywords/stat4-phosphorylation","display_name":"STAT4 phosphorylation","score":0.9049999713897705},{"id":"https://openalex.org/keywords/interferon-gamma-production","display_name":"IFN-gamma production","score":0.8799999952316284},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7789999842643738}],"concepts":[{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.6831732988357544},{"id":"https://openalex.org/C65439459","wikidata":"https://www.wikidata.org/wiki/Q14914618","display_name":"STAT4","level":5,"score":0.6223394274711609},{"id":"https://openalex.org/C62478195","wikidata":"https://www.wikidata.org/wiki/Q828130","display_name":"Signal transduction","level":2,"score":0.595161497592926},{"id":"https://openalex.org/C149532602","wikidata":"https://www.wikidata.org/wiki/Q2409681","display_name":"Interleukin 12","level":4,"score":0.5077089071273804},{"id":"https://openalex.org/C170493617","wikidata":"https://www.wikidata.org/wiki/Q208467","display_name":"Receptor","level":2,"score":0.4994630813598633},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.47665244340896606},{"id":"https://openalex.org/C15152581","wikidata":"https://www.wikidata.org/wiki/Q7833966","display_name":"Transduction (biophysics)","level":2,"score":0.47446972131729126},{"id":"https://openalex.org/C70883133","wikidata":"https://www.wikidata.org/wiki/Q7318316","display_name":"Reversion","level":4,"score":0.4627426266670227},{"id":"https://openalex.org/C2778690821","wikidata":"https://www.wikidata.org/wiki/Q212354","display_name":"Cytokine","level":2,"score":0.4555869698524475},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.42930692434310913},{"id":"https://openalex.org/C127716648","wikidata":"https://www.wikidata.org/wiki/Q104053","display_name":"Phenotype","level":3,"score":0.3999228775501251},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.39447951316833496},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.3767874836921692},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.2528955936431885},{"id":"https://openalex.org/C154317977","wikidata":"https://www.wikidata.org/wiki/Q376266","display_name":"Cytotoxic T cell","level":3,"score":0.21287453174591064},{"id":"https://openalex.org/C202751555","wikidata":"https://www.wikidata.org/wiki/Q221681","display_name":"In vitro","level":2,"score":0.07723724842071533},{"id":"https://openalex.org/C2778277574","wikidata":"https://www.wikidata.org/wiki/Q665327","display_name":"stat","level":4,"score":0.0},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0},{"id":"https://openalex.org/C2778923194","wikidata":"https://www.wikidata.org/wiki/Q14912019","display_name":"STAT3","level":3,"score":0.0}],"mesh":[{"descriptor_ui":"D001424","descriptor_name":"Bacterial Infections","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D001424","descriptor_name":"Bacterial Infections","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D001424","descriptor_name":"Bacterial Infections","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":true},{"descriptor_ui":"D002460","descriptor_name":"Cell Line","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004268","descriptor_name":"DNA-Binding Proteins","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007371","descriptor_name":"Interferon-gamma","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D007371","descriptor_name":"Interferon-gamma","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D007378","descriptor_name":"Interleukins","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D007378","descriptor_name":"Interleukins","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D007378","descriptor_name":"Interleukins","qualifier_ui":"Q000494","qualifier_name":"pharmacology","is_major_topic":true},{"descriptor_ui":"D010766","descriptor_name":"Phosphorylation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D012190","descriptor_name":"Retroviridae","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D013601","descriptor_name":"T-Lymphocytes","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D015398","descriptor_name":"Signal Transduction","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D015534","descriptor_name":"Trans-Activators","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018123","descriptor_name":"Receptors, Interleukin","qualifier_ui":"Q000032","qualifier_name":"analysis","is_major_topic":false},{"descriptor_ui":"D018123","descriptor_name":"Receptors, Interleukin","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D018123","descriptor_name":"Receptors, Interleukin","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D018664","descriptor_name":"Interleukin-12","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D018664","descriptor_name":"Interleukin-12","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D018664","descriptor_name":"Interleukin-12","qualifier_ui":"Q000494","qualifier_name":"pharmacology","is_major_topic":false},{"descriptor_ui":"D050797","descriptor_name":"STAT4 Transcription Factor","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D053707","descriptor_name":"Receptors, Interleukin-12","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D053759","descriptor_name":"Interleukin-23","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D053760","descriptor_name":"Interleukin-23 Subunit p19","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":2,"locations":[{"id":"doi:10.1016/j.ymthe.2004.02.024","doi":"https://doi.org/10.1016/j.ymthe.2004.02.024","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.ymthe.2004.02.024","pdf_url":"http://www.cell.com/article/S1525001604001005/pdf","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":"pmid:15194056","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/15194056","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:10.1016/j.ymthe.2004.02.024","doi":"https://doi.org/10.1016/j.ymthe.2004.02.024","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.ymthe.2004.02.024","pdf_url":"http://www.cell.com/article/S1525001604001005/pdf","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":[{"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":0.7200000286102295,"display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[{"id":"https://openalex.org/F4320331528","display_name":"Ministero dell'Universit\u00e0 e della Ricerca","ror":"https://ror.org/0341vw408"},{"id":"https://openalex.org/F4320335310","display_name":"Fondation BNP Paribas","ror":"https://ror.org/02v616z87"}],"has_content":{"grobid_xml":false,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W1970617299.pdf"},"referenced_works_count":30,"referenced_works":["https://openalex.org/W206621073","https://openalex.org/W1623924764","https://openalex.org/W1847326477","https://openalex.org/W1908249423","https://openalex.org/W1936243145","https://openalex.org/W1973985508","https://openalex.org/W1979876156","https://openalex.org/W1984342091","https://openalex.org/W1988825974","https://openalex.org/W1991524518","https://openalex.org/W2009219530","https://openalex.org/W2018995171","https://openalex.org/W2029255163","https://openalex.org/W2037187119","https://openalex.org/W2039700479","https://openalex.org/W2049390045","https://openalex.org/W2065601509","https://openalex.org/W2077256612","https://openalex.org/W2080385201","https://openalex.org/W2087905758","https://openalex.org/W2109830199","https://openalex.org/W2121717907","https://openalex.org/W2122696789","https://openalex.org/W2135254713","https://openalex.org/W2137254849","https://openalex.org/W2140897891","https://openalex.org/W2154218327","https://openalex.org/W2162867382","https://openalex.org/W2171382646","https://openalex.org/W2280192492"],"related_works":["https://openalex.org/W7217893068","https://openalex.org/W1600259869","https://openalex.org/W1970536217","https://openalex.org/W1989690639","https://openalex.org/W2014381801","https://openalex.org/W2154218327","https://openalex.org/W2165396917","https://openalex.org/W4234387910","https://openalex.org/W1936243145","https://openalex.org/W7024297476"],"abstract_inverted_index":{"Genetic":[0],"deficiency":[1,152],"of":[2,17,28,46,60,82,86,95,98,105,112,145,166],"human":[3],"IL-12":[4,29,115],"receptor":[5],"beta1":[6],"chain":[7],"(IL-12Rbeta1)":[8],"results":[9,23,139],"in":[10,109,128],"increased":[11],"vulnerability":[12],"to":[13,54,130],"weakly":[14],"pathogenic":[15],"strains":[16],"Mycobacteria":[18],"and":[19,30,48,107,123,159],"Salmonella.":[20],"This":[21],"phenotype":[22],"from":[24,90,148],"the":[25,69,80,99,103,110,142,161],"combined":[26],"lack":[27],"IL-23":[31,131],"signaling":[32,116],"as":[33,118],"both":[34],"cytokine":[35],"receptors":[36],"share":[37],"IL-12Rbeta1.":[38,96],"Such":[39],"infections":[40,55],"can":[41,52,153],"be":[42,154],"treated":[43],"by":[44,63,120,156],"administration":[45],"antibiotics":[47],"IFN-gamma;":[49],"however,":[50],"patients":[51,91,149],"succumb":[53],"despite":[56],"these":[57],"treatments.":[58],"Reversion":[59],"patients'":[61],"susceptibility":[62],"corrective":[64],"gene":[65,84,136,157,167],"transfer":[66,158],"could":[67],"prevent":[68],"infectious":[70],"episodes,":[71],"thus":[72],"providing":[73],"a":[74,113],"beneficial":[75],"alternative.":[76],"We":[77],"therefore":[78],"evaluated":[79],"feasibility":[81],"retroviral-mediated":[83],"correction":[85],"T":[87,146],"cells":[88,147],"obtained":[89],"carrying":[92,150],"\"null\"":[93],"mutations":[94],"Transduction":[97],"IL-12Rbeta1":[100,106,151],"cDNA":[101],"restored":[102],"expression":[104],"resulted":[108],"reconstitution":[111],"functional":[114],"pathway,":[117],"demonstrated":[119],"STAT4":[121],"phosphorylation":[122],"IFN-gamma":[124,126],"production.":[125],"production":[127],"response":[129],"was":[132],"also":[133],"corrected":[134,155],"after":[135],"transfer.":[137],"These":[138],"indicate":[140],"that":[141],"biological":[143],"defects":[144],"form":[160],"basis":[162],"for":[163,169],"further":[164],"development":[165],"therapy":[168],"this":[170],"disease.":[171]},"counts_by_year":[{"year":2019,"cited_by_count":1},{"year":2016,"cited_by_count":1},{"year":2013,"cited_by_count":1}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2016-06-24T00:00:00"}