{"id":"https://openalex.org/W2159530833","doi":"https://doi.org/10.1016/s0140-6736(04)17590-9","title":"Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector","display_name":"Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector","publication_year":2004,"publication_date":"2004-12-01","ids":{"openalex":"https://openalex.org/W2159530833","doi":"https://doi.org/10.1016/s0140-6736(04)17590-9","mag":"2159530833","pmid":"https://pubmed.ncbi.nlm.nih.gov/15610804"},"language":"en","primary_location":{"id":"doi:10.1016/s0140-6736(04)17590-9","doi":"https://doi.org/10.1016/s0140-6736(04)17590-9","is_oa":false,"landing_page_url":"https://doi.org/10.1016/s0140-6736(04)17590-9","pdf_url":null,"source":{"id":"https://openalex.org/S49861241","display_name":"The Lancet","issn_l":"0099-5355","issn":["0099-5355","0140-6736","1474-547X"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","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":"The Lancet","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/A5072838648","display_name":"Hubert Bobby Gaspar","orcid":"https://orcid.org/0000-0001-6700-7213","observed_orcids":["https://orcid.org/0000-0001-6700-7213"]},"institutions":[{"id":"https://openalex.org/I125671874","display_name":"National Health Service","ror":"https://ror.org/02wnqcb97","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I125671874","https://openalex.org/I1311074006"]},{"id":"https://openalex.org/I2800129641","display_name":"Great Ormond Street Hospital","ror":"https://ror.org/00zn2c847","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I2800129641","https://openalex.org/I2800349819"]},{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"H Bobby Gaspar","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]},{"raw_affiliation_string":"Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK","institution_ids":["https://openalex.org/I125671874","https://openalex.org/I2800129641"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108466764","display_name":"Kathryn L. Parsley","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Kathryn L Parsley","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5103246215","display_name":"Steven J. Howe","orcid":"https://orcid.org/0000-0002-8656-8670","observed_orcids":["https://orcid.org/0000-0002-8656-8670","https://orcid.org/0000-0003-2940-2874"]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Steven Howe","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Doug King","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Doug King","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5085859205","display_name":"Kimberly C Gilmour","orcid":"https://orcid.org/0000-0003-0285-3958","observed_orcids":["https://orcid.org/0000-0003-0285-3958"]},"institutions":[{"id":"https://openalex.org/I125671874","display_name":"National Health Service","ror":"https://ror.org/02wnqcb97","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I125671874","https://openalex.org/I1311074006"]},{"id":"https://openalex.org/I2800129641","display_name":"Great Ormond Street Hospital","ror":"https://ror.org/00zn2c847","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I2800129641","https://openalex.org/I2800349819"]},{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Kimberly C Gilmour","raw_affiliation_strings":["Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK","Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK","institution_ids":["https://openalex.org/I125671874","https://openalex.org/I2800129641"]},{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5112714686","display_name":"Joanna C. Sinclair","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Joanna Sinclair","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5001146963","display_name":"Gaby S. Brouns","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Gaby Brouns","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5060449046","display_name":"Manfred Schmidt","orcid":"https://orcid.org/0009-0001-9632-0599","observed_orcids":["https://orcid.org/0009-0001-9632-0599"]},"institutions":[{"id":"https://openalex.org/I161046081","display_name":"University of Freiburg","ror":"https://ror.org/0245cg223","country_code":"DE","type":"education","lineage":["https://openalex.org/I161046081"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Manfred Schmidt","raw_affiliation_strings":["Department of Internal Medicine and Institute of Molecular Medicine and Cell Research, University of Freiburg, Freiburg, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Internal Medicine and Institute of Molecular Medicine and Cell Research, University of Freiburg, Freiburg, Germany","institution_ids":["https://openalex.org/I161046081"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5084073390","display_name":"Christof V. Kalle","orcid":"https://orcid.org/0000-0001-9221-3297","observed_orcids":["https://orcid.org/0000-0001-9221-3297"]},"institutions":[{"id":"https://openalex.org/I1285204247","display_name":"Cincinnati Children's Hospital Medical Center","ror":"https://ror.org/01hcyya48","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1285204247"]},{"id":"https://openalex.org/I161046081","display_name":"University of Freiburg","ror":"https://ror.org/0245cg223","country_code":"DE","type":"education","lineage":["https://openalex.org/I161046081"]}],"countries":["DE","US"],"is_corresponding":false,"raw_author_name":"Christof Von Kalle","raw_affiliation_strings":["Department of Internal Medicine and Institute of Molecular Medicine and Cell Research, University of Freiburg, Freiburg, Germany","Division of Experimental Hematology, Cincinnati Children's Research Foundation, Cincinnati, OH, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Internal Medicine and Institute of Molecular Medicine and Cell Research, University of Freiburg, Freiburg, Germany","institution_ids":["https://openalex.org/I161046081"]},{"raw_affiliation_string":"Division of Experimental Hematology, Cincinnati Children's Research Foundation, Cincinnati, OH, USA","institution_ids":["https://openalex.org/I1285204247"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5042091545","display_name":"Torben Barington","orcid":"https://orcid.org/0000-0001-6968-148X","observed_orcids":["https://orcid.org/0000-0001-6968-148X"]},"institutions":[{"id":"https://openalex.org/I2801498763","display_name":"Odense University Hospital","ror":"https://ror.org/00ey0ed83","country_code":"DK","type":"funder","lineage":["https://openalex.org/I2801498763"]}],"countries":["DK"],"is_corresponding":false,"raw_author_name":"Torben Barington","raw_affiliation_strings":["Department of Clinical Immunology, Odense University Hospital, Odense, Denmark"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Clinical Immunology, Odense University Hospital, Odense, Denmark","institution_ids":["https://openalex.org/I2801498763"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5090286433","display_name":"Marianne Antonius Jakobsen","orcid":"https://orcid.org/0000-0003-2352-9276","observed_orcids":["https://orcid.org/0000-0003-2352-9276"]},"institutions":[{"id":"https://openalex.org/I2801498763","display_name":"Odense University Hospital","ror":"https://ror.org/00ey0ed83","country_code":"DK","type":"funder","lineage":["https://openalex.org/I2801498763"]}],"countries":["DK"],"is_corresponding":false,"raw_author_name":"Marianne A Jakobsen","raw_affiliation_strings":["Department of Clinical Immunology, Odense University Hospital, Odense, Denmark"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Clinical Immunology, Odense University Hospital, Odense, Denmark","institution_ids":["https://openalex.org/I2801498763"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5058434776","display_name":"Hans Ole Christensen","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2801498763","display_name":"Odense University Hospital","ror":"https://ror.org/00ey0ed83","country_code":"DK","type":"funder","lineage":["https://openalex.org/I2801498763"]}],"countries":["DK"],"is_corresponding":false,"raw_author_name":"Hans O Christensen","raw_affiliation_strings":["Department of Paediatrics, Odense University Hospital, Odense Denmark"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Paediatrics, Odense University Hospital, Odense Denmark","institution_ids":["https://openalex.org/I2801498763"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5053058921","display_name":"Abdulaziz Al Ghonaium","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2800965796","display_name":"King Faisal Specialist Hospital & Research Centre","ror":"https://ror.org/05n0wgt02","country_code":"SA","type":"healthcare","lineage":["https://openalex.org/I2800965796"]}],"countries":["SA"],"is_corresponding":false,"raw_author_name":"Abdulaziz Al Ghonaium","raw_affiliation_strings":["Department of Paediatric Allergy/Immunology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Paediatric Allergy/Immunology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia","institution_ids":["https://openalex.org/I2800965796"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5104048344","display_name":"Harry N White","orcid":"https://orcid.org/0000-0001-8186-7789","observed_orcids":["https://orcid.org/0000-0001-8186-7789"]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Harry N White","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108530506","display_name":"John Lewis Smith","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2801388914","display_name":"Southampton General Hospital","ror":"https://ror.org/011cztj49","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I2801388914","https://openalex.org/I2802965805"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"John L Smith","raw_affiliation_strings":["Department of Immunology, Southampton General Hospital, Southampton, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Immunology, Southampton General Hospital, Southampton, UK","institution_ids":["https://openalex.org/I2801388914"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108483409","display_name":"Roland J. Levinsky","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Roland J Levinsky","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5073699632","display_name":"Robin R. Ali","orcid":"https://orcid.org/0000-0003-3126-6517","observed_orcids":["https://orcid.org/0000-0003-3126-6517"]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Robin R Ali","raw_affiliation_strings":["Department of Molecular Genetics, Institute of Ophthalmology, University College London, London, UK","Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Molecular Genetics, Institute of Ophthalmology, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]},{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5080762141","display_name":"Christine Kinnon","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Christine Kinnon","raw_affiliation_strings":["Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5015856478","display_name":"Adrian James Thrasher","orcid":"https://orcid.org/0000-0002-6097-6115","observed_orcids":["https://orcid.org/0000-0002-6097-6115"]},"institutions":[{"id":"https://openalex.org/I125671874","display_name":"National Health Service","ror":"https://ror.org/02wnqcb97","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I125671874","https://openalex.org/I1311074006"]},{"id":"https://openalex.org/I2800129641","display_name":"Great Ormond Street Hospital","ror":"https://ror.org/00zn2c847","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I2800129641","https://openalex.org/I2800349819"]},{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":true,"raw_author_name":"Adrian J Thrasher","raw_affiliation_strings":["Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK","Molecular Immunology Unit, Institute of Child Health, University College London, London, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK","institution_ids":["https://openalex.org/I125671874","https://openalex.org/I2800129641"]},{"raw_affiliation_string":"Molecular Immunology Unit, Institute of Child Health, University College London, London, UK","institution_ids":["https://openalex.org/I45129253"]}]}],"institutions":[],"countries_distinct_count":5,"institutions_distinct_count":8,"corresponding_author_ids":["https://openalex.org/A5015856478"],"corresponding_institution_ids":["https://openalex.org/I125671874","https://openalex.org/I2800129641","https://openalex.org/I45129253"],"apc_list":{"value":7890,"currency":"USD","value_usd":7890},"apc_paid":null,"fwci":24.7387,"has_fulltext":false,"cited_by_count":689,"citation_normalized_percentile":{"value":0.99825149,"is_in_top_1_percent":true,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":97,"max":100},"biblio":{"volume":"364","issue":"9452","first_page":"2181","last_page":"2187"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9965999722480774,"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":0.9965999722480774,"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":0.0008999999845400453,"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/T11491","display_name":"CAR-T cell therapy research","score":0.00019999999494757503,"subfield":{"id":"https://openalex.org/subfields/2730","display_name":"Oncology"},"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/x-linked-severe-combined-immunodeficiency","display_name":"X-linked severe combined immunodeficiency","score":0.9900000095367432},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7870000004768372},{"id":"https://openalex.org/keywords/common-gamma-chain","display_name":"common gamma chain","score":0.628000020980835},{"id":"https://openalex.org/keywords/autologous-bone-marrow-stem-cells","display_name":"autologous bone marrow stem cells","score":0.7089999914169312},{"id":"https://openalex.org/keywords/t-cell-reconstitution","display_name":"T cell reconstitution","score":0.5860000252723694},{"id":"https://openalex.org/keywords/humoral-immunity","display_name":"humoral immunity","score":0.8980000019073486},{"id":"https://openalex.org/keywords/tcr-repertoire","display_name":"T cell receptor repertoire","score":0.7260000109672546}],"concepts":[{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.7026107311248779},{"id":"https://openalex.org/C2779468541","wikidata":"https://www.wikidata.org/wiki/Q1334408","display_name":"Severe combined immunodeficiency","level":3,"score":0.6952669024467468},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.693810760974884},{"id":"https://openalex.org/C2777607303","wikidata":"https://www.wikidata.org/wiki/Q641307","display_name":"Immunodeficiency","level":3,"score":0.5734280943870544},{"id":"https://openalex.org/C98654944","wikidata":"https://www.wikidata.org/wiki/Q14885185","display_name":"Common gamma chain","level":4,"score":0.49381813406944275},{"id":"https://openalex.org/C2780007613","wikidata":"https://www.wikidata.org/wiki/Q546523","display_name":"Bone marrow","level":2,"score":0.483602911233902},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.48236554861068726},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.470650851726532},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.44962531328201294},{"id":"https://openalex.org/C159654299","wikidata":"https://www.wikidata.org/wiki/Q79460","display_name":"Antibody","level":2,"score":0.4393068552017212},{"id":"https://openalex.org/C2779341262","wikidata":"https://www.wikidata.org/wiki/Q182581","display_name":"Immunity","level":3,"score":0.42414480447769165},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.34575366973876953},{"id":"https://openalex.org/C207001950","wikidata":"https://www.wikidata.org/wiki/Q141124","display_name":"In vivo","level":2,"score":0.20102888345718384},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.13288208842277527},{"id":"https://openalex.org/C187316574","wikidata":"https://www.wikidata.org/wiki/Q423698","display_name":"Interleukin 10","level":3,"score":0.11738544702529907},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.0},{"id":"https://openalex.org/C150903083","wikidata":"https://www.wikidata.org/wiki/Q7108","display_name":"Biotechnology","level":1,"score":0.0},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D001854","descriptor_name":"Bone Marrow Cells","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D001854","descriptor_name":"Bone Marrow Cells","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D001854","descriptor_name":"Bone Marrow Cells","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007109","descriptor_name":"Immunity","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007109","descriptor_name":"Immunity","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007109","descriptor_name":"Immunity","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007136","descriptor_name":"Immunoglobulins","qualifier_ui":"Q000097","qualifier_name":"blood","is_major_topic":false},{"descriptor_ui":"D007136","descriptor_name":"Immunoglobulins","qualifier_ui":"Q000097","qualifier_name":"blood","is_major_topic":false},{"descriptor_ui":"D007136","descriptor_name":"Immunoglobulins","qualifier_ui":"Q000097","qualifier_name":"blood","is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007959","descriptor_name":"Lymphocyte Culture Test, Mixed","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007959","descriptor_name":"Lymphocyte Culture Test, Mixed","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007959","descriptor_name":"Lymphocyte Culture Test, Mixed","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008213","descriptor_name":"Lymphocyte Activation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008213","descriptor_name":"Lymphocyte Activation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008213","descriptor_name":"Lymphocyte Activation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009154","descriptor_name":"Mutation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009154","descriptor_name":"Mutation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009154","descriptor_name":"Mutation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D013601","descriptor_name":"T-Lymphocytes","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D013601","descriptor_name":"T-Lymphocytes","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D013601","descriptor_name":"T-Lymphocytes","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D014161","descriptor_name":"Transduction, Genetic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D014161","descriptor_name":"Transduction, Genetic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D014161","descriptor_name":"Transduction, Genetic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D016026","descriptor_name":"Bone Marrow Transplantation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016026","descriptor_name":"Bone Marrow Transplantation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016026","descriptor_name":"Bone Marrow Transplantation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D016511","descriptor_name":"Severe Combined Immunodeficiency","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D017988","descriptor_name":"Gammaretrovirus","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D017988","descriptor_name":"Gammaretrovirus","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D017988","descriptor_name":"Gammaretrovirus","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018952","descriptor_name":"Antigens, CD34","qualifier_ui":"Q000032","qualifier_name":"analysis","is_major_topic":false},{"descriptor_ui":"D018952","descriptor_name":"Antigens, CD34","qualifier_ui":"Q000032","qualifier_name":"analysis","is_major_topic":false},{"descriptor_ui":"D018952","descriptor_name":"Antigens, CD34","qualifier_ui":"Q000032","qualifier_name":"analysis","is_major_topic":false},{"descriptor_ui":"D020395","descriptor_name":"Receptors, Interleukin-7","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D020395","descriptor_name":"Receptors, Interleukin-7","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D020395","descriptor_name":"Receptors, Interleukin-7","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D040181","descriptor_name":"Genetic Diseases, X-Linked","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D040181","descriptor_name":"Genetic Diseases, X-Linked","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D040181","descriptor_name":"Genetic Diseases, X-Linked","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D040181","descriptor_name":"Genetic Diseases, X-Linked","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D040181","descriptor_name":"Genetic Diseases, X-Linked","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D040181","descriptor_name":"Genetic Diseases, X-Linked","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D053631","descriptor_name":"Interleukin Receptor Common gamma Subunit","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D053631","descriptor_name":"Interleukin Receptor Common gamma Subunit","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D053631","descriptor_name":"Interleukin Receptor Common gamma Subunit","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":6,"locations":[{"id":"doi:10.1016/s0140-6736(04)17590-9","doi":"https://doi.org/10.1016/s0140-6736(04)17590-9","is_oa":false,"landing_page_url":"https://doi.org/10.1016/s0140-6736(04)17590-9","pdf_url":null,"source":{"id":"https://openalex.org/S49861241","display_name":"The Lancet","issn_l":"0099-5355","issn":["0099-5355","0140-6736","1474-547X"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","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":"The Lancet","raw_type":"journal-article"},{"id":"pmid:15610804","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/15610804","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":"Lancet (London, England)","raw_type":"Journal Article"},{"id":"pmh:oai:eprints.ucl.ac.uk.OAI2:110473","doi":null,"is_oa":false,"landing_page_url":"http://discovery.ucl.ac.uk/110473/","pdf_url":null,"source":{"id":"https://openalex.org/S4306400024","display_name":"UCL Discovery (University College London)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I45129253","host_organization_name":"University College London","host_organization_lineage":["https://openalex.org/I45129253"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Lancet , 364 (9452) pp. 2181-2187. (2004)","raw_type":"Article"},{"id":"pmh:oai:eprints.ucl.ac.uk.OAI2:8077","doi":null,"is_oa":false,"landing_page_url":"http://discovery.ucl.ac.uk/8077/","pdf_url":null,"source":{"id":"https://openalex.org/S4306400024","display_name":"UCL Discovery (University College London)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I45129253","host_organization_name":"University College London","host_organization_lineage":["https://openalex.org/I45129253"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"LANCET , 364 (9452) 2181 - 2187. (2004)","raw_type":"Article"},{"id":"pmh:oai:repository.library.georgetown.edu:10822/505878","doi":null,"is_oa":false,"landing_page_url":"http://hdl.handle.net/10822/505878","pdf_url":null,"source":{"id":"https://openalex.org/S4306402467","display_name":"DigitalGeorgetown (Georgetown University Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I4210145449","host_organization_name":"Center for Security and Emerging Technology","host_organization_lineage":["https://openalex.org/I4210145449"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"278655","raw_type":null},{"id":"pmh:oai:sdu.dk:openaire_cris_publications/6c8c1f70-ed84-11db-821c-000ea68e967b","doi":null,"is_oa":false,"landing_page_url":"https://portal.findresearcher.sdu.dk/da/publications/6c8c1f70-ed84-11db-821c-000ea68e967b","pdf_url":null,"source":{"id":"https://openalex.org/S4306400423","display_name":"University of Southern Denmark Research Portal (University of Southern Denmark)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I177969490","host_organization_name":"University of Southern Denmark","host_organization_lineage":["https://openalex.org/I177969490"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Gaspar, H B, Parsley, K L, Howe, S, King, D, Gilmour, K C, Sinclair, J, Brouns, G, Schmidt, M, Von Kalle, C, Barington, T, Jakobsen, M A, Christensen, H O, Al Ghonaium, A, White, H N, Smith, J L, Levinsky, R J, Ali, R R, Kinnon, C & Trasher, A J 2004, 'Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.', The Lancet, vol. 364, no. 9452, pp. 2181-2187. https://doi.org/10.1016/S0140-6736(04)17590-9","raw_type":"info:eu-repo/semantics/article"}],"best_oa_location":null,"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","score":0.9940000176429749,"display_name":"Good health and well-being"}],"sustainable_development_goals_aurora":[{"id":"https://metadata.un.org/sdg/3","score":0.8600000143051147,"display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G2071472662","display_name":"Analysis of the diversity of the B-cell memory compartment","funder_award_id":"062578","funder_id":"https://openalex.org/F4320311904","funder_display_name":"Wellcome Trust"}],"funders":[{"id":"https://openalex.org/F4320311904","display_name":"Wellcome Trust","ror":"https://ror.org/029chgv08"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":22,"referenced_works":["https://openalex.org/W193981811","https://openalex.org/W1971799885","https://openalex.org/W1980132147","https://openalex.org/W1985513262","https://openalex.org/W2000468629","https://openalex.org/W2045705588","https://openalex.org/W2083345299","https://openalex.org/W2083685529","https://openalex.org/W2091268360","https://openalex.org/W2091972495","https://openalex.org/W2100890363","https://openalex.org/W2107278930","https://openalex.org/W2116700965","https://openalex.org/W2137440275","https://openalex.org/W2152461163","https://openalex.org/W2171801248","https://openalex.org/W2268875976","https://openalex.org/W2323613689","https://openalex.org/W2330288813","https://openalex.org/W2533802729","https://openalex.org/W3151452517","https://openalex.org/W6607943104"],"related_works":["https://openalex.org/W2291465919","https://openalex.org/W1996422725","https://openalex.org/W2122340353","https://openalex.org/W2026187066","https://openalex.org/W2095073287","https://openalex.org/W2912603519","https://openalex.org/W2032196358","https://openalex.org/W4310378707","https://openalex.org/W2347300262","https://openalex.org/W119695473"],"abstract_inverted_index":null,"counts_by_year":[{"year":2026,"cited_by_count":3},{"year":2025,"cited_by_count":12},{"year":2024,"cited_by_count":7},{"year":2023,"cited_by_count":15},{"year":2022,"cited_by_count":12},{"year":2021,"cited_by_count":17},{"year":2020,"cited_by_count":25},{"year":2019,"cited_by_count":26},{"year":2018,"cited_by_count":16},{"year":2017,"cited_by_count":22},{"year":2016,"cited_by_count":20},{"year":2015,"cited_by_count":30},{"year":2014,"cited_by_count":33},{"year":2013,"cited_by_count":28},{"year":2012,"cited_by_count":42}],"updated_date":"2026-10-16T09:59:22.235173","created_date":"2025-10-10T00:00:00"}