{"id":"https://openalex.org/W1234938885","doi":"https://doi.org/10.1038/mt.2015.154","title":"Functional Restoration of gp91phox-Oxidase Activity by BAC Transgenesis and Gene Targeting in X-linked Chronic Granulomatous Disease iPSCs","display_name":"Functional Restoration of gp91phox-Oxidase Activity by BAC Transgenesis and Gene Targeting in X-linked Chronic Granulomatous Disease iPSCs","publication_year":2015,"publication_date":"2015-08-28","ids":{"openalex":"https://openalex.org/W1234938885","doi":"https://doi.org/10.1038/mt.2015.154","mag":"1234938885","pmid":"https://pubmed.ncbi.nlm.nih.gov/26316390","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/4886927"},"language":"en","primary_location":{"id":"doi:10.1038/mt.2015.154","doi":"https://doi.org/10.1038/mt.2015.154","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2015.154","pdf_url":null,"source":{"id":"https://openalex.org/S51596929","display_name":"Molecular Therapy","issn_l":"1525-0016","issn":["1525-0016","1525-0024"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-3","ki-jl-2","medline","norway-1","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://doi.org/10.1038/mt.2015.154","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5018420158","display_name":"Magdalena Laugsch","orcid":"https://orcid.org/0009-0006-6079-7017","observed_orcids":["https://orcid.org/0009-0006-6079-7017"]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Magdalena Laugsch","raw_affiliation_strings":["Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5002051588","display_name":"Maria Rostovskaya","orcid":"https://orcid.org/0000-0003-1187-8663","observed_orcids":["https://orcid.org/0000-0003-1187-8663"]},"institutions":[{"id":"https://openalex.org/I4405272450","display_name":"Biotechnologisches Zentrum","ror":"https://ror.org/046b28292","country_code":"DE","type":"facility","lineage":["https://openalex.org/I4405272450","https://openalex.org/I4405279746","https://openalex.org/I78650965"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Maria Rostovskaya","raw_affiliation_strings":["Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4405272450","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5036570118","display_name":"Sergiy Velychko","orcid":"https://orcid.org/0000-0002-6227-3966","observed_orcids":["https://orcid.org/0000-0002-6227-3966"]},"institutions":[{"id":"https://openalex.org/I4405272450","display_name":"Biotechnologisches Zentrum","ror":"https://ror.org/046b28292","country_code":"DE","type":"facility","lineage":["https://openalex.org/I4405272450","https://openalex.org/I4405279746","https://openalex.org/I78650965"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Sergiy Velychko","raw_affiliation_strings":["Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4405272450","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5042676481","display_name":"Cornelia Richter","orcid":"https://orcid.org/0000-0003-4559-5696","observed_orcids":["https://orcid.org/0000-0003-4559-5696"]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Cornelia Richter","raw_affiliation_strings":["Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5082495006","display_name":"Ariane Zimmer","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Ariane Zimmer","raw_affiliation_strings":["Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5037027253","display_name":"Barbara Klink","orcid":"https://orcid.org/0000-0002-8753-8238","observed_orcids":["https://orcid.org/0000-0002-8753-8238"]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Barbara Klink","raw_affiliation_strings":["Institute for Clinical Genetics, Faculty of Medicine Carl Gustav Carus, TU, Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Clinical Genetics, Faculty of Medicine Carl Gustav Carus, TU, Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5046141686","display_name":"Evelin Schr\u00f6ck","orcid":"https://orcid.org/0000-0002-3377-1704","observed_orcids":["https://orcid.org/0000-0002-3377-1704"]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Evelin Schr\u00f6ck","raw_affiliation_strings":["Institute for Clinical Genetics, Faculty of Medicine Carl Gustav Carus, TU, Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Institute for Clinical Genetics, Faculty of Medicine Carl Gustav Carus, TU, Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5031315923","display_name":"Michael Haase","orcid":"https://orcid.org/0000-0002-5897-0320","observed_orcids":["https://orcid.org/0000-0002-5897-0320"]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Michael Haase","raw_affiliation_strings":["Department of Pediatric Surgery, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatric Surgery, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5049581826","display_name":"Katrin Neumann","orcid":"https://orcid.org/0000-0002-3858-3171","observed_orcids":["https://orcid.org/0000-0002-3858-3171","https://orcid.org/0000-0002-3989-8095"]},"institutions":[{"id":"https://openalex.org/I4405272450","display_name":"Biotechnologisches Zentrum","ror":"https://ror.org/046b28292","country_code":"DE","type":"facility","lineage":["https://openalex.org/I4405272450","https://openalex.org/I4405279746","https://openalex.org/I78650965"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Katrin Neumann","raw_affiliation_strings":["Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4405272450","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5101559293","display_name":"Sebastian Thieme","orcid":"https://orcid.org/0000-0002-8565-5051","observed_orcids":["https://orcid.org/0000-0002-8565-5051"]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Sebastian Thieme","raw_affiliation_strings":["Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5010045339","display_name":"Joachim Roesler","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"Joachim Roesler","raw_affiliation_strings":["Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5026466385","display_name":"Sebastian Brenner","orcid":"https://orcid.org/0000-0002-3666-7128","observed_orcids":["https://orcid.org/0000-0002-3666-7128"]},"institutions":[{"id":"https://openalex.org/I4210162051","display_name":"University Hospital Carl Gustav Carus","ror":"https://ror.org/04za5zm41","country_code":"DE","type":"healthcare","lineage":["https://openalex.org/I4210162051"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":true,"raw_author_name":"Sebastian Brenner","raw_affiliation_strings":["Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, University Clinic Carl Gustav Carus, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I78650965"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5087616533","display_name":"Konstantinos Anastassiadis","orcid":"https://orcid.org/0000-0002-9814-0559","observed_orcids":["https://orcid.org/0000-0002-9814-0559"]},"institutions":[{"id":"https://openalex.org/I4405272450","display_name":"Biotechnologisches Zentrum","ror":"https://ror.org/046b28292","country_code":"DE","type":"facility","lineage":["https://openalex.org/I4405272450","https://openalex.org/I4405279746","https://openalex.org/I78650965"]},{"id":"https://openalex.org/I78650965","display_name":"Technische Universit\u00e4t Dresden","ror":"https://ror.org/042aqky30","country_code":"DE","type":"education","lineage":["https://openalex.org/I78650965"]}],"countries":["DE"],"is_corresponding":true,"raw_author_name":"Konstantinos Anastassiadis","raw_affiliation_strings":["Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Engineering, BIOTEC, TU Dresden, Dresden, Germany","institution_ids":["https://openalex.org/I4405272450","https://openalex.org/I78650965"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5026466385","https://openalex.org/A5087616533"],"corresponding_institution_ids":["https://openalex.org/I4210162051","https://openalex.org/I4405272450","https://openalex.org/I78650965"],"apc_list":{"value":4500,"currency":"USD","value_usd":4500},"apc_paid":{"value":4500,"currency":"USD","value_usd":4500},"fwci":0.923,"has_fulltext":false,"cited_by_count":24,"citation_normalized_percentile":{"value":0.70716902,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":88,"max":99},"biblio":{"volume":"24","issue":"4","first_page":"812","last_page":"822"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10878","display_name":"CRISPR and Genetic Engineering","score":0.4146000146865845,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10878","display_name":"CRISPR and Genetic Engineering","score":0.4146000146865845,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.24950000643730164,"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/T10505","display_name":"Pluripotent Stem Cells Research","score":0.21570000052452087,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/x-linked-chronic-granulomatous-disease","display_name":"X-linked chronic granulomatous disease","score":0.9940000176429749},{"id":"https://openalex.org/keywords/gp91phox","display_name":"gp91phox","score":0.9309999942779541},{"id":"https://openalex.org/keywords/cybb","display_name":"CYBB","score":0.8849999904632568},{"id":"https://openalex.org/keywords/nadph-oxidase","display_name":"NADPH oxidase","score":0.9810000061988831},{"id":"https://openalex.org/keywords/induced-pluripotent-stem-cells","display_name":"induced pluripotent stem cells","score":0.9610000252723694},{"id":"https://openalex.org/keywords/bac-transgenesis","display_name":"BAC transgenesis","score":0.8370000123977661},{"id":"https://openalex.org/keywords/lentiviral-reprogramming","display_name":"lentiviral reprogramming","score":0.718999981880188},{"id":"https://openalex.org/keywords/keratinocytes","display_name":"keratinocytes","score":0.8180000185966492},{"id":"https://openalex.org/keywords/myeloid-differentiation","display_name":"myeloid differentiation","score":0.890999972820282},{"id":"https://openalex.org/keywords/autologous-cell-therapy","display_name":"autologous cell therapy","score":0.800000011920929},{"id":"https://openalex.org/keywords/monogenic-diseases","display_name":"monogenic disorders","score":0.4429999887943268}],"concepts":[{"id":"https://openalex.org/C2778175462","wikidata":"https://www.wikidata.org/wiki/Q2165663","display_name":"Chronic granulomatous disease","level":2,"score":0.9234789609909058},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.6179788112640381},{"id":"https://openalex.org/C73247094","wikidata":"https://www.wikidata.org/wiki/Q2790163","display_name":"Transgenesis","level":5,"score":0.5711923241615295},{"id":"https://openalex.org/C77255625","wikidata":"https://www.wikidata.org/wiki/Q402093","display_name":"Reprogramming","level":3,"score":0.5615127682685852},{"id":"https://openalex.org/C107459253","wikidata":"https://www.wikidata.org/wiki/Q674925","display_name":"Induced pluripotent stem cell","level":4,"score":0.5270105600357056},{"id":"https://openalex.org/C111829913","wikidata":"https://www.wikidata.org/wiki/Q211083","display_name":"Gene targeting","level":3,"score":0.48457300662994385},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.47237303853034973},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.44222503900527954},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3785138428211212},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.32374727725982666},{"id":"https://openalex.org/C145103041","wikidata":"https://www.wikidata.org/wiki/Q1151519","display_name":"Embryonic stem cell","level":3,"score":0.14869913458824158},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.10877951979637146},{"id":"https://openalex.org/C2994278967","wikidata":"https://www.wikidata.org/wiki/Q7314318","display_name":"Reproductive technology","level":4,"score":0.1069057285785675},{"id":"https://openalex.org/C87073359","wikidata":"https://www.wikidata.org/wiki/Q33196","display_name":"Embryogenesis","level":3,"score":0.0}],"mesh":[{"descriptor_ui":"D000074662","descriptor_name":"NADPH Oxidase 2","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002454","descriptor_name":"Cell Differentiation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002478","descriptor_name":"Cells, Cultured","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":"D006105","descriptor_name":"Granulomatous Disease, Chronic","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D006105","descriptor_name":"Granulomatous Disease, Chronic","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":true},{"descriptor_ui":"D006105","descriptor_name":"Granulomatous Disease, Chronic","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008562","descriptor_name":"Membrane Glycoproteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D008562","descriptor_name":"Membrane Glycoproteins","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D009154","descriptor_name":"Mutation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D015603","descriptor_name":"Keratinocytes","qualifier_ui":"Q000166","qualifier_name":"cytology","is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D018390","descriptor_name":"Gene Targeting","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D019255","descriptor_name":"NADPH Oxidases","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D019255","descriptor_name":"NADPH Oxidases","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D022202","descriptor_name":"Chromosomes, Artificial, Bacterial","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D057026","descriptor_name":"Induced Pluripotent Stem Cells","qualifier_ui":"Q000201","qualifier_name":"enzymology","is_major_topic":true}],"locations_count":4,"locations":[{"id":"doi:10.1038/mt.2015.154","doi":"https://doi.org/10.1038/mt.2015.154","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2015.154","pdf_url":null,"source":{"id":"https://openalex.org/S51596929","display_name":"Molecular Therapy","issn_l":"1525-0016","issn":["1525-0016","1525-0024"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-3","ki-jl-2","medline","norway-1","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy","raw_type":"journal-article"},{"id":"pmh:oai:europepmc.org:3859439","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/4886927","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":"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":null,"raw_type":"Text"},{"id":"pmh:oai:pubmedcentral.nih.gov:4886927","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/4886927","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":"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":"Mol Ther","raw_type":"Text"},{"id":"pmid:26316390","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/26316390","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular therapy : the journal of the American Society of Gene Therapy","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1038/mt.2015.154","doi":"https://doi.org/10.1038/mt.2015.154","is_oa":true,"landing_page_url":"https://doi.org/10.1038/mt.2015.154","pdf_url":null,"source":{"id":"https://openalex.org/S51596929","display_name":"Molecular Therapy","issn_l":"1525-0016","issn":["1525-0016","1525-0024"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-3","ki-jl-2","medline","norway-1","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Molecular Therapy","raw_type":"journal-article"},"sustainable_development_goals":[{"score":0.98580002784729,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G2899563092","display_name":"Erstellung und Korrektur Patienten-spezifischer iPS Zellen zur Therapie der Septischen Granulomatose (CGD)","funder_award_id":"01GN1007","funder_id":"https://openalex.org/F4320321114","funder_display_name":"Bundesministerium f\u00fcr Bildung und Forschung"}],"funders":[{"id":"https://openalex.org/F4320321114","display_name":"Bundesministerium f\u00fcr Bildung und Forschung","ror":"https://ror.org/04pz7b180"},{"id":"https://openalex.org/F4320335238","display_name":"Staatssekretariat f\u00fcr Bildung, Forschung und Innovation","ror":"https://ror.org/01kw63t33"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":57,"referenced_works":["https://openalex.org/W1966530560","https://openalex.org/W1982179068","https://openalex.org/W1984433385","https://openalex.org/W1986439862","https://openalex.org/W1987111575","https://openalex.org/W1990707605","https://openalex.org/W1992838550","https://openalex.org/W2008995006","https://openalex.org/W2012044302","https://openalex.org/W2013940523","https://openalex.org/W2014870062","https://openalex.org/W2021230455","https://openalex.org/W2021312195","https://openalex.org/W2023161233","https://openalex.org/W2023456870","https://openalex.org/W2036632122","https://openalex.org/W2037479635","https://openalex.org/W2038753879","https://openalex.org/W2040065556","https://openalex.org/W2051140434","https://openalex.org/W2053988054","https://openalex.org/W2058021805","https://openalex.org/W2059187839","https://openalex.org/W2066707761","https://openalex.org/W2070942486","https://openalex.org/W2073377349","https://openalex.org/W2073793908","https://openalex.org/W2075136524","https://openalex.org/W2076026160","https://openalex.org/W2077659966","https://openalex.org/W2079527931","https://openalex.org/W2081970575","https://openalex.org/W2082223061","https://openalex.org/W2083731850","https://openalex.org/W2083837591","https://openalex.org/W2083871413","https://openalex.org/W2086663462","https://openalex.org/W2090424915","https://openalex.org/W2095442157","https://openalex.org/W2100465572","https://openalex.org/W2102264927","https://openalex.org/W2102718460","https://openalex.org/W2104978512","https://openalex.org/W2106485710","https://openalex.org/W2118918764","https://openalex.org/W2129583958","https://openalex.org/W2135108891","https://openalex.org/W2136426539","https://openalex.org/W2144938275","https://openalex.org/W2154177867","https://openalex.org/W2158771952","https://openalex.org/W2160331473","https://openalex.org/W2160429342","https://openalex.org/W2259938310","https://openalex.org/W2333111179","https://openalex.org/W4254125610","https://openalex.org/W4310912077"],"related_works":["https://openalex.org/W2023456870","https://openalex.org/W1486348543","https://openalex.org/W2001138357","https://openalex.org/W2410546905","https://openalex.org/W2135108891","https://openalex.org/W3752704","https://openalex.org/W2006913836","https://openalex.org/W2054821808","https://openalex.org/W1487614847","https://openalex.org/W1970223002"],"abstract_inverted_index":{"Chronic":[0],"granulomatous":[1],"disease":[2],"(CGD)":[3],"is":[4,31],"an":[5,141,157],"inherited":[6],"immunodeficiency,":[7],"caused":[8],"by":[9,140],"the":[10,36,124,137,150,161,169],"inability":[11],"of":[12,27,42,49,97,112,123,136,163,171],"neutrophils":[13],"to":[14,33,107,116],"produce":[15],"functional":[16,134],"NADPH":[17,43],"oxidase":[18,142],"required":[19],"for":[20,46,168],"fighting":[21],"microbial":[22],"infections.":[23],"The":[24],"X-linked":[25],"form":[26],"CGD":[28,50],"(X-CGD),":[29],"which":[30],"due":[32],"mutations":[34],"in":[35,103,133,145,176],"CYBB":[37],"(gp91phox)":[38],"gene,":[39],"a":[40,64,91],"component":[41],"oxidase,":[44],"accounts":[45],"about":[47],"two-thirds":[48],"cases.":[51],"We":[52],"derived":[53],"induced":[54],"pluripotent":[55],"stem":[56],"cells":[57,167],"(iPSCs)":[58],"from":[59,105],"X-CGD":[60,146,172],"patient":[61],"keratinocytes":[62],"using":[63,88],"Flp":[65],"recombinase":[66],"excisable":[67],"lentiviral":[68],"reprogramming":[69],"vector.":[70],"For":[71],"restoring":[72],"gp91phox":[73,138],"function,":[74],"we":[75,155],"applied":[76],"two":[77],"strategies:":[78],"transposon-mediated":[79],"bacterial":[80],"artificial":[81],"chromosome":[82],"(BAC)":[83],"transgenesis":[84,128],"and":[85,100,129,173],"gene":[86,130],"targeting":[87,131],"vectors":[89],"with":[90,120],"fixed":[92],"5'":[93],"homology":[94],"arm":[95],"(HA)":[96],"8":[98],"kb":[99],"3'HA":[101],"varying":[102],"size":[104,122],"30":[106],"80":[108],"kb.":[109],"High":[110],"efficiency":[111],"homologous":[113],"recombination":[114],"(up":[115],"22%)":[117],"was":[118],"observed":[119],"increased":[121],"3'HA.":[125],"Both,":[126],"BAC":[127],"resulted":[132],"restoration":[135],"measured":[139],"activity":[143],"assay":[144],"iPSCs":[147],"differentiated":[148],"into":[149],"myeloid":[151],"lineage.":[152],"In":[153],"conclusion,":[154],"delivered":[156],"important":[158],"milestone":[159],"towards":[160],"use":[162],"genetically":[164],"corrected":[165],"autologous":[166],"treatment":[170],"monogenic":[174],"diseases":[175],"general.":[177]},"counts_by_year":[{"year":2025,"cited_by_count":3},{"year":2022,"cited_by_count":2},{"year":2021,"cited_by_count":2},{"year":2020,"cited_by_count":2},{"year":2019,"cited_by_count":7},{"year":2018,"cited_by_count":1},{"year":2017,"cited_by_count":3},{"year":2016,"cited_by_count":3},{"year":2015,"cited_by_count":1}],"updated_date":"2026-11-07T11:25:07.637180","created_date":"2016-06-24T00:00:00"}