{"id":"https://openalex.org/W1968381323","doi":"https://doi.org/10.1038/mt.2011.312","title":"Nonmyeloablative Conditioning Regimen to Increase Engraftment of Gene-modified Hematopoietic Stem Cells in Young Rhesus Monkeys","display_name":"Nonmyeloablative Conditioning Regimen to Increase Engraftment of Gene-modified Hematopoietic Stem Cells in Young Rhesus Monkeys","publication_year":2012,"publication_date":"2012-01-31","ids":{"openalex":"https://openalex.org/W1968381323","doi":"https://doi.org/10.1038/mt.2011.312","mag":"1968381323","pmid":"https://pubmed.ncbi.nlm.nih.gov/22294147","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/3345994"},"language":"en","primary_location":{"id":"doi:10.1038/mt.2011.312","doi":"https://doi.org/10.1038/mt.2011.312","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2011.312","pdf_url":"http://www.cell.com/article/S1525001616319438/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/S1525001616319438/pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5109176248","display_name":"Alice F. Tarantal","orcid":"https://orcid.org/0000-0002-8153-1094","observed_orcids":["https://orcid.org/0000-0002-8153-1094"]},"institutions":[{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Alice F Tarantal","raw_affiliation_strings":["Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, California National Primate Research Center, Davis, California, USA","Department of Cell Biology and Human Anatomy, University of California, Davis, California, USA","Department of Pediatrics, University of California, Davis, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, California National Primate Research Center, Davis, California, USA","institution_ids":[]},{"raw_affiliation_string":"Department of Cell Biology and Human Anatomy, University of California, Davis, California, USA","institution_ids":["https://openalex.org/I84218800"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Davis, California, USA","institution_ids":["https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5103485492","display_name":"Francesca Giannoni","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I161318765","display_name":"University of California, Los Angeles","ror":"https://ror.org/046rm7j60","country_code":"US","type":"education","lineage":["https://openalex.org/I161318765"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Francesca Giannoni","raw_affiliation_strings":["Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5070699826","display_name":"C Chang I Lee","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"C Chang I Lee","raw_affiliation_strings":["Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, California National Primate Research Center, Davis, California, USA","Department of Cell Biology and Human Anatomy, University of California, Davis, California, USA","Department of Pediatrics, University of California, Davis, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, California National Primate Research Center, Davis, California, USA","institution_ids":[]},{"raw_affiliation_string":"Department of Cell Biology and Human Anatomy, University of California, Davis, California, USA","institution_ids":["https://openalex.org/I84218800"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Davis, California, USA","institution_ids":["https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5049636647","display_name":"Jennifer Wherley","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I161318765","display_name":"University of California, Los Angeles","ror":"https://ror.org/046rm7j60","country_code":"US","type":"education","lineage":["https://openalex.org/I161318765"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jennifer Wherley","raw_affiliation_strings":["Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5015912578","display_name":"Teiko Sumiyoshi","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I161318765","display_name":"University of California, Los Angeles","ror":"https://ror.org/046rm7j60","country_code":"US","type":"education","lineage":["https://openalex.org/I161318765"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Teiko Sumiyoshi","raw_affiliation_strings":["Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5103973700","display_name":"Michele L. Martinez","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Michele Martinez","raw_affiliation_strings":["Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, California National Primate Research Center, Davis, California, USA","Department of Cell Biology and Human Anatomy, University of California, Davis, California, USA","Department of Pediatrics, University of California, Davis, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, California National Primate Research Center, Davis, California, USA","institution_ids":[]},{"raw_affiliation_string":"Department of Cell Biology and Human Anatomy, University of California, Davis, California, USA","institution_ids":["https://openalex.org/I84218800"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Davis, California, USA","institution_ids":["https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Christoph A Kahl","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I102507086","display_name":"Oregon National Primate Research Center","ror":"https://ror.org/05fcfqq67","country_code":"US","type":"facility","lineage":["https://openalex.org/I102507086"]},{"id":"https://openalex.org/I161318765","display_name":"University of California, Los Angeles","ror":"https://ror.org/046rm7j60","country_code":"US","type":"education","lineage":["https://openalex.org/I161318765"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Christoph A Kahl","raw_affiliation_strings":["Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA","Present address: Oregon Health and Science University, Oregon National Primate Research Center, Beaverton, Oregon, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]},{"raw_affiliation_string":"Present address: Oregon Health and Science University, Oregon National Primate Research Center, Beaverton, Oregon, USA","institution_ids":["https://openalex.org/I102507086"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5070990818","display_name":"David A. Elashoff","orcid":"https://orcid.org/0000-0002-5865-8580","observed_orcids":["https://orcid.org/0000-0002-5865-8580"]},"institutions":[{"id":"https://openalex.org/I161318765","display_name":"University of California, Los Angeles","ror":"https://ror.org/046rm7j60","country_code":"US","type":"education","lineage":["https://openalex.org/I161318765"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"David Elashoff","raw_affiliation_strings":["Department of Medicine Statistics Core, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Medicine Statistics Core, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5002462952","display_name":"Stan G. Louie","orcid":"https://orcid.org/0000-0001-5941-6690","observed_orcids":["https://orcid.org/0000-0001-5941-6690"]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Stan G Louie","raw_affiliation_strings":["Titus Family Department of Clinical Pharmacy and Pharmaceutical Economics and Policy, University of Southern California School of Pharmacy, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Titus Family Department of Clinical Pharmacy and Pharmaceutical Economics and Policy, University of Southern California School of Pharmacy, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1174212"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5050971129","display_name":"Donald B. Kohn","orcid":"https://orcid.org/0000-0003-1840-6087","observed_orcids":["https://orcid.org/0000-0003-1840-6087"]},"institutions":[{"id":"https://openalex.org/I161318765","display_name":"University of California, Los Angeles","ror":"https://ror.org/046rm7j60","country_code":"US","type":"education","lineage":["https://openalex.org/I161318765"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Donald B Kohn","raw_affiliation_strings":["Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]},{"raw_affiliation_string":"Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I161318765"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":4,"corresponding_author_ids":["https://openalex.org/A5050971129"],"corresponding_institution_ids":["https://openalex.org/I161318765"],"apc_list":{"value":4500,"currency":"USD","value_usd":4500},"apc_paid":{"value":4500,"currency":"USD","value_usd":4500},"fwci":1.1986,"has_fulltext":false,"cited_by_count":27,"citation_normalized_percentile":{"value":0.77077935,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":89,"max":98},"biblio":{"volume":"20","issue":"5","first_page":"1033","last_page":"1045"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9932000041007996,"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":0.9932000041007996,"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/T10345","display_name":"Hematopoietic Stem Cell Transplantation","score":0.004000000189989805,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"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/T12929","display_name":"Animal Genetics and Reproduction","score":0.00019999999494757503,"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"}}],"keywords":[{"id":"https://openalex.org/keywords/nonmyeloablative-conditioning","display_name":"nonmyeloablative conditioning","score":0.984000027179718},{"id":"https://openalex.org/keywords/hematopoietic-engraftment","display_name":"hematopoietic engraftment","score":0.9520000219345093},{"id":"https://openalex.org/keywords/busulfan","display_name":"busulfan","score":0.7900000214576721},{"id":"https://openalex.org/keywords/fludarabine","display_name":"fludarabine","score":1.0},{"id":"https://openalex.org/keywords/immunosuppression","display_name":"immunosuppression","score":0.6819999814033508},{"id":"https://openalex.org/keywords/lentiviral-vectors","display_name":"lentiviral vectors","score":0.8679999709129333},{"id":"https://openalex.org/keywords/simian-immunodeficiency-virus","display_name":"simian immunodeficiency virus","score":0.8399999737739563},{"id":"https://openalex.org/keywords/rhesus-macaques","display_name":"rhesus monkeys","score":0.7239999771118164},{"id":"https://openalex.org/keywords/cd34-cells","display_name":"CD34+ cells","score":0.6909999847412109},{"id":"https://openalex.org/keywords/gfp-reporter","display_name":"GFP reporter","score":0.6840000152587891},{"id":"https://openalex.org/keywords/transgene-persistence","display_name":"transgene persistence","score":0.5730000138282776},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7639999985694885}],"concepts":[{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.7330427765846252},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.6840966939926147},{"id":"https://openalex.org/C109159458","wikidata":"https://www.wikidata.org/wiki/Q919283","display_name":"Haematopoiesis","level":3,"score":0.6336566805839539},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.6064586043357849},{"id":"https://openalex.org/C102230213","wikidata":"https://www.wikidata.org/wiki/Q430117","display_name":"Transgene","level":3,"score":0.5913679003715515},{"id":"https://openalex.org/C28328180","wikidata":"https://www.wikidata.org/wiki/Q48196","display_name":"Stem cell","level":2,"score":0.5607276558876038},{"id":"https://openalex.org/C2780611847","wikidata":"https://www.wikidata.org/wiki/Q348922","display_name":"Busulfan","level":4,"score":0.5386794209480286},{"id":"https://openalex.org/C2780007613","wikidata":"https://www.wikidata.org/wiki/Q546523","display_name":"Bone marrow","level":2,"score":0.51643967628479},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.47537437081336975},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.4622160792350769},{"id":"https://openalex.org/C2778086970","wikidata":"https://www.wikidata.org/wiki/Q514525","display_name":"Hematopoietic stem cell","level":4,"score":0.42388278245925903},{"id":"https://openalex.org/C142613039","wikidata":"https://www.wikidata.org/wiki/Q272983","display_name":"Green fluorescent protein","level":3,"score":0.4106799066066742},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.3608373999595642},{"id":"https://openalex.org/C2777408962","wikidata":"https://www.wikidata.org/wiki/Q1128050","display_name":"Hematopoietic stem cell transplantation","level":3,"score":0.32581794261932373},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.19237303733825684},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.18841248750686646},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.06353873014450073}],"mesh":[{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000831","descriptor_name":"Animals, Newborn","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002066","descriptor_name":"Busulfan","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006085","descriptor_name":"Graft Survival","qualifier_ui":"Q000187","qualifier_name":"drug effects","is_major_topic":false},{"descriptor_ui":"D006085","descriptor_name":"Graft Survival","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":true},{"descriptor_ui":"D006412","descriptor_name":"Hematopoietic Stem Cells","qualifier_ui":"Q000276","qualifier_name":"immunology","is_major_topic":true},{"descriptor_ui":"D007108","descriptor_name":"Immune Tolerance","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007166","descriptor_name":"Immunosuppressive Agents","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D008253","descriptor_name":"Macaca mulatta","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":"D014740","descriptor_name":"Vidarabine","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D014740","descriptor_name":"Vidarabine","qualifier_ui":"Q000031","qualifier_name":"analogs & derivatives","is_major_topic":false},{"descriptor_ui":"D015302","descriptor_name":"Simian Immunodeficiency Virus","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D016086","descriptor_name":"Lentivirus","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D017930","descriptor_name":"Genes, Reporter","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018380","descriptor_name":"Hematopoietic Stem Cell Transplantation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D019076","descriptor_name":"Transgenes","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D019172","descriptor_name":"Transplantation Conditioning","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D049452","descriptor_name":"Green Fluorescent Proteins","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":4,"locations":[{"id":"doi:10.1038/mt.2011.312","doi":"https://doi.org/10.1038/mt.2011.312","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2011.312","pdf_url":"http://www.cell.com/article/S1525001616319438/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":"pmh:oai:europepmc.org:2637988","doi":null,"is_oa":true,"landing_page_url":"http://europepmc.org/articles/PMC3345994","pdf_url":null,"source":{"id":"https://openalex.org/S4306400806","display_name":"Europe PMC (PubMed Central)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1303153112","host_organization_name":"European Bioinformatics Institute","host_organization_lineage":["https://openalex.org/I1303153112"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"","raw_type":"Text"},{"id":"pmh:oai:pubmedcentral.nih.gov:3345994","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/3345994","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:22294147","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/22294147","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.2011.312","doi":"https://doi.org/10.1038/mt.2011.312","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2011.312","pdf_url":"http://www.cell.com/article/S1525001616319438/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":0.9763000011444092,"display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1013472931","display_name":"California National Primate Research Center","funder_award_id":"P51RR000169","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G2113117208","display_name":"Gene Therapy Using Hematopoietic Stem Cells","funder_award_id":"P01HL073104","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G3276713972","display_name":"UCLA Clinical and Translational Science Institute","funder_award_id":"UL1TR000124","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G7153988826","display_name":"Center for Fetal Gene Transfer for Heart, Lung, and Blood Diseases","funder_award_id":"R24HL085794","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G8522415837","display_name":"In Vivo ADA Gene Delivery for the Treatment of SCID","funder_award_id":"R01AI074043","funder_id":"https://openalex.org/F4320337355","funder_display_name":"National Institute of Allergy and Infectious Diseases"}],"funders":[{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320337338","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26"},{"id":"https://openalex.org/F4320337355","display_name":"National Institute of Allergy and Infectious Diseases","ror":"https://ror.org/043z4tv69"},{"id":"https://openalex.org/F4320337375","display_name":"National Center for Research Resources","ror":"https://ror.org/04pw6fb54"},{"id":"https://openalex.org/F4320337472","display_name":"National Center for Advancing Translational Sciences","ror":"https://ror.org/04pw6fb54"}],"has_content":{"pdf":true,"grobid_xml":false},"content_urls":{"pdf":"https://content.openalex.org/works/W1968381323.pdf"},"referenced_works_count":43,"referenced_works":["https://openalex.org/W55776777","https://openalex.org/W70627539","https://openalex.org/W230061309","https://openalex.org/W1972896863","https://openalex.org/W1974916809","https://openalex.org/W2005815716","https://openalex.org/W2009279477","https://openalex.org/W2010899405","https://openalex.org/W2017304478","https://openalex.org/W2018570885","https://openalex.org/W2023480073","https://openalex.org/W2032287795","https://openalex.org/W2041245805","https://openalex.org/W2044649483","https://openalex.org/W2044875199","https://openalex.org/W2048164516","https://openalex.org/W2066271426","https://openalex.org/W2082607509","https://openalex.org/W2088494611","https://openalex.org/W2091268360","https://openalex.org/W2092976340","https://openalex.org/W2105358704","https://openalex.org/W2107278930","https://openalex.org/W2113409179","https://openalex.org/W2121444412","https://openalex.org/W2127433915","https://openalex.org/W2132505135","https://openalex.org/W2137442186","https://openalex.org/W2137632760","https://openalex.org/W2139028184","https://openalex.org/W2140880458","https://openalex.org/W2145586276","https://openalex.org/W2159530833","https://openalex.org/W2163229914","https://openalex.org/W2246750959","https://openalex.org/W2289043455","https://openalex.org/W2304337710","https://openalex.org/W2396162351","https://openalex.org/W2415551722","https://openalex.org/W3151452517","https://openalex.org/W4234839810","https://openalex.org/W4290201891","https://openalex.org/W6680027276"],"related_works":["https://openalex.org/W2948621850","https://openalex.org/W4384699717","https://openalex.org/W2113568719","https://openalex.org/W2317467567","https://openalex.org/W1989528483","https://openalex.org/W2162217603","https://openalex.org/W269397754","https://openalex.org/W4416867525","https://openalex.org/W2071317527","https://openalex.org/W2010899405"],"abstract_inverted_index":{"Immune":[0],"responses":[1,45,147,191],"to":[2,7,46,64,68,76,148,175,192],"transgene":[3,47,74],"products":[4],"may":[5,28],"lead":[6],"rejection":[8],"of":[9,22,115,117,130,157,179,185],"transduced":[10,83,186],"cells,":[11,131,159],"limiting":[12],"successful":[13],"gene":[14,197],"therapy":[15],"for":[16],"genetic":[17],"diseases.":[18],"While":[19],"moderate":[20],"dosages":[21],"chemotherapeutic":[23],"agents":[24],"such":[25],"as":[26],"busulfan":[27,55],"increase":[29],"hematopoietic":[30],"stem":[31],"cells":[32,70,118,162,193],"(HSC)":[33],"engraftment,":[34],"they":[35],"are":[36],"not":[37,42,164],"immune":[38,44,66,146,190],"suppressive":[39],"and":[40,102,132,144,188],"do":[41],"abrogate":[43],"products.":[48],"Studies":[49],"focused":[50],"on":[51,182],"nonmyeloablative":[52],"conditioning":[53,180],"with":[54,137,153],"\u00b1":[56],"fludarabine":[57],"in":[58,84,151],"a":[59,72,96,169,195],"clinically":[60,170],"relevant":[61,171],"monkey":[62],"model":[63,174],"induce":[65],"suppression":[67],"allow":[69],"expressing":[71,194],"foreign":[73,196],"product":[75],"persist.":[77],"Bone":[78],"marrow":[79],"CD34(+)":[80],"HSC":[81,187],"were":[82,135,163],"two":[85],"equal":[86],"fractions":[87],"using":[88],"simian":[89],"immunodeficiency":[90],"virus":[91],"(SIV)-based":[92],"lentiviral":[93],"vectors":[94],"carrying":[95],"nonexpressed":[97],"DNA":[98],"sequence":[99],"tag":[100],"(NoN)":[101],"the":[103,120,154,177,183,189],"green":[104],"fluorescent":[105],"protein":[106],"(GFP)":[107],"reporter":[108],"gene.":[109],"Post-transplant":[110],"there":[111],"was":[112,140],"no":[113,133],"evidence":[114],"elimination":[116],"containing":[119],"potentially":[121],"immunogenic":[122],"GFP":[123,149],"gene;":[124],"several":[125],"recipients":[126,152],"had":[127],"stable":[128],"persistence":[129],"differences":[134],"detected":[136],"fludarabine,":[138],"which":[139],"rapidly":[141],"cleared.":[142],"Antibodies":[143],"cellular":[145],"developed":[150],"highest":[155],"levels":[156],"GFP-marked":[158],"although":[160],"these":[161],"eliminated.":[165],"These":[166],"studies":[167],"establish":[168],"pediatric":[172],"primate":[173],"assess":[176],"effects":[178],"regimens":[181],"engraftment":[184],"product.":[198]},"counts_by_year":[{"year":2026,"cited_by_count":1},{"year":2025,"cited_by_count":1},{"year":2024,"cited_by_count":1},{"year":2022,"cited_by_count":4},{"year":2020,"cited_by_count":2},{"year":2019,"cited_by_count":4},{"year":2018,"cited_by_count":1},{"year":2017,"cited_by_count":2},{"year":2016,"cited_by_count":3},{"year":2015,"cited_by_count":1},{"year":2014,"cited_by_count":4},{"year":2013,"cited_by_count":1},{"year":2012,"cited_by_count":2}],"updated_date":"2026-11-06T12:05:46.655746","created_date":"2016-06-24T00:00:00"}
