{"id":"https://openalex.org/W262255493","doi":null,"title":"Episomal expression of wild-type CFTR corrects cAMP-dependent chloride transport in respiratory epithelial cells.","display_name":"Episomal expression of wild-type CFTR corrects cAMP-dependent chloride transport in respiratory epithelial cells.","publication_year":1996,"publication_date":"1996-05-01","ids":{"openalex":"https://openalex.org/W262255493","mag":"262255493","pmid":"https://pubmed.ncbi.nlm.nih.gov/9156804"},"language":"en","primary_location":{"id":"pmid:9156804","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/9156804","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":"Gene therapy","raw_type":"Journal Article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["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/A5054583491","display_name":"Dachuan Lei","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I180670191","display_name":"University of California, San Francisco","ror":"https://ror.org/043mz5j54","country_code":"US","type":"education","lineage":["https://openalex.org/I180670191"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"D C Lei","raw_affiliation_strings":["Cardiovascular Research Institute, University of California, San Francisco, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Cardiovascular Research Institute, University of California, San Francisco, CA, USA","institution_ids":["https://openalex.org/I180670191"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5051912802","display_name":"Karl Kunzelmann","orcid":"https://orcid.org/0000-0001-6222-4593","observed_orcids":["https://orcid.org/0000-0001-6222-4593","https://orcid.org/0000-0002-4583-7037"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"K Kunzelmann","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5082791032","display_name":"Thomas Christian Koslowsky","orcid":"https://orcid.org/0000-0003-1793-6780","observed_orcids":["https://orcid.org/0000-0003-1793-6780"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"T Koslowsky","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5049016785","display_name":"Michael J. Yezzi","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"M J Yezzi","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5077396552","display_name":"L. C. Escobar","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"L C Escobar","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111498400","display_name":"Zhidong Xu","orcid":"https://orcid.org/0000-0002-8109-1471","observed_orcids":["https://orcid.org/0000-0002-8109-1471"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Z Xu","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5013804712","display_name":"Aaron R. Ellison","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"A R Ellison","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5008426210","display_name":"Johanna M. Rommens","orcid":"https://orcid.org/0000-0001-8732-825X","observed_orcids":["https://orcid.org/0000-0001-8732-825X"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"J M Rommens","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5112025484","display_name":"Tsui Lc","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Tsui L-C","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5055325270","display_name":"Mark L. Tykocinski","orcid":"https://orcid.org/0000-0002-3520-5300","observed_orcids":["https://orcid.org/0000-0002-3520-5300"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"M Tykocinski","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"last","author":{"id":"https://openalex.org/A5046074910","display_name":"Dieter C. Gruenert","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"D C Gruenert","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":4.3251,"has_fulltext":false,"cited_by_count":55,"citation_normalized_percentile":{"value":0.9383841,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":88,"max":93},"biblio":{"volume":"3","issue":"5","first_page":"427","last_page":"36"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10665","display_name":"Cystic Fibrosis Research Advances","score":0.989300012588501,"subfield":{"id":"https://openalex.org/subfields/2740","display_name":"Pulmonary and Respiratory Medicine"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T10665","display_name":"Cystic Fibrosis Research Advances","score":0.989300012588501,"subfield":{"id":"https://openalex.org/subfields/2740","display_name":"Pulmonary and Respiratory Medicine"},"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/T10613","display_name":"Virus-based gene therapy research","score":0.006200000178068876,"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/T10725","display_name":"RNA Interference and Gene Delivery","score":0.00039999998989515007,"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/cystic-fibrosis","display_name":"cystic fibrosis","score":0.9760000109672546},{"id":"https://openalex.org/keywords/cftr","display_name":"CFTR","score":0.9789999723434448},{"id":"https://openalex.org/keywords/cystic-fibrosis-transmembrane-conductance-regulator","display_name":"cystic fibrosis transmembrane conductance regulator","score":0.9319999814033508},{"id":"https://openalex.org/keywords/airway-epithelial-cells","display_name":"airway epithelial cells","score":0.7099999785423279},{"id":"https://openalex.org/keywords/rous-sarcoma-virus-long-terminal-repeat","display_name":"Rous sarcoma virus long terminal repeat","score":0.8209999799728394},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7239999771118164},{"id":"https://openalex.org/keywords/plasmid-copy-number","display_name":"plasmid copy number","score":0.7009999752044678}],"concepts":[{"id":"https://openalex.org/C2778428886","wikidata":"https://www.wikidata.org/wiki/Q420470","display_name":"Cystic fibrosis transmembrane conductance regulator","level":3,"score":0.8292242288589478},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.6449456214904785},{"id":"https://openalex.org/C2776938444","wikidata":"https://www.wikidata.org/wiki/Q178194","display_name":"Cystic fibrosis","level":2,"score":0.630133867263794},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.5483744740486145},{"id":"https://openalex.org/C150194340","wikidata":"https://www.wikidata.org/wiki/Q26972","display_name":"Gene expression","level":3,"score":0.5151702165603638},{"id":"https://openalex.org/C54009773","wikidata":"https://www.wikidata.org/wiki/Q1429031","display_name":"Transfection","level":3,"score":0.4995889663696289},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.44440770149230957},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.3286365270614624},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.31340932846069336},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.14141348004341125},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.1344221830368042}],"mesh":[{"descriptor_ui":"D000242","descriptor_name":"Cyclic AMP","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":true},{"descriptor_ui":"D002478","descriptor_name":"Cells, Cultured","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002712","descriptor_name":"Chlorides","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":true},{"descriptor_ui":"D003550","descriptor_name":"Cystic Fibrosis","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D003550","descriptor_name":"Cystic Fibrosis","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D004848","descriptor_name":"Epithelium","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D005786","descriptor_name":"Gene Expression Regulation","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":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D010957","descriptor_name":"Plasmids","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":true},{"descriptor_ui":"D017136","descriptor_name":"Ion Transport","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D019005","descriptor_name":"Cystic Fibrosis Transmembrane Conductance Regulator","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D019005","descriptor_name":"Cystic Fibrosis Transmembrane Conductance Regulator","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false}],"locations_count":3,"locations":[{"id":"pmid:9156804","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/9156804","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":"Gene therapy","raw_type":"Journal Article"},{"id":"pmh:oai:hub.hku.hk:10722/44300","doi":null,"is_oa":false,"landing_page_url":"http://hdl.handle.net/10722/44300","pdf_url":null,"source":{"id":"https://openalex.org/S4377196271","display_name":"The HKU Scholars Hub (University of Hong Kong)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I889458895","host_organization_name":"University of Hong Kong","host_organization_lineage":["https://openalex.org/I889458895"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"","raw_type":"Article"},{"id":"mag:262255493","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/9156804","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":null,"is_accepted":false,"is_published":false,"raw_source_name":"PubMed","raw_type":null}],"best_oa_location":null,"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":0.9537000060081482}],"study_designs":[],"awards":[{"id":"https://openalex.org/G4185238686","display_name":"GENE THERAPY CORE--CYSTIC FIBROSIS &NON-CF GENETIC DIS","funder_award_id":"P30DK047766","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"}],"funders":[{"id":"https://openalex.org/F4320337357","display_name":"National Institute of Diabetes and Digestive and Kidney Diseases","ror":"https://ror.org/00adh9b73"}],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W1991604425","https://openalex.org/W2037872751","https://openalex.org/W2016532191","https://openalex.org/W1981015157","https://openalex.org/W2079791074","https://openalex.org/W2036921663","https://openalex.org/W1993993814","https://openalex.org/W2146538727","https://openalex.org/W2409683519","https://openalex.org/W2140407450"],"abstract_inverted_index":{"The":[0,164],"isolation":[1,48],"of":[2,38,49,66,94,110,151,166,177,187,191,206,210,218,224,265,273,277],"the":[3,7,21,24,28,47,50,53,64,95,108,111,175,222,225,263,266,274,281],"gene":[4,68],"responsible":[5],"for":[6,92,137,184],"Cl-":[8,97,138,237,254],"ion":[9,139],"transport":[10,98,140,238,255],"defect":[11,99],"in":[12,130,199,221,256,270],"cystic":[13,54],"fibrosis":[14,55],"(CF)":[15],"has":[16,60],"provided":[17],"important":[18],"information":[19],"about":[20],"relationship":[22],"between":[23],"disease":[25],"pathology":[26],"and":[27,31,40,79,145,148,239],"underlying":[29],"genetic":[30],"biochemical":[32],"mechanisms.":[33],"In":[34],"addition,":[35],"new":[36],"areas":[37],"investigation":[39],"therapy":[41,69],"are":[42],"now":[43],"possible.":[44],"Most":[45],"notably,":[46],"CF":[51],"gene,":[52],"transmembrane":[56],"conductance":[57],"regulator":[58],"(CFTR)":[59],"been":[61],"led":[62],"to":[63,76,168,171,201,229,251,296],"development":[65],"different":[67],"strategies.":[70],"To":[71],"circumvent":[72],"possible":[73,250],"complications":[74],"due":[75],"insertional":[77],"mutagenesis":[78],"virally":[80],"induced":[81],"immune":[82],"responses,":[83],"we":[84],"have":[85],"employed":[86],"Epstein-Barr":[87],"virus":[88,114],"(EBV)-based":[89],"expression":[90,105,186],"vectors":[91],"correction":[93],"cAMP-dependent":[96,131,153,236,253],"associated":[100],"with":[101,215,293],"CF.":[102],"A":[103],"CFTR-containing":[104],"construct":[106],"under":[107],"regulation":[109],"Rous":[112],"sarcoma":[113],"(RSV)":[115],"long":[116],"terminal":[117],"repeat":[118],"(LTR)":[119],"(pREP5/CFTR)":[120],"was":[121,159,248,286],"introduced":[122],"into":[123],"transformed":[124],"human":[125],"airway":[126],"epithelial":[127],"cells":[128,134,181,259],"defective":[129],"Cl-transport.":[132],"Transfected":[133],"were":[135,182,243],"assayed":[136],"by":[141,161,260],"(36)Cl-":[142],"efflux,":[143],"SPQ,":[144],"patch":[146],"clamp":[147],"showed":[149,195],"restoration":[150],"intact":[152],"transport.":[154],"CFTR":[155,178,188,219,241,267],"transcription":[156],"from":[157,288],"pREP5/CFTR":[158,282],"detected":[160],"Northern":[162],"hybridization.":[163],"level":[165,176],"response":[167],"agonists":[169],"appeared":[170],"be":[172],"dependent":[173],"on":[174],"expression.":[179],"When":[180],"tested":[183],"functional":[185],"after":[189,208,302],"removal":[190,209],"selection":[192,234],"pressure,":[193],"they":[194],"a":[196,204,216,271],"continuous":[197,294],"decrease":[198,213],"responsiveness":[200],"forskolin":[202],"as":[203],"function":[205],"time":[207],"selection.":[211],"This":[212],"correlated":[214],"loss":[217,223],"mRNA":[220,242],"PREP5/CFTR.":[226],"After":[227],"12":[228],"15":[230],"weeks":[231,304],"growth":[232],"without":[233,305],"both":[235],"plasmid-derived":[240],"not":[244],"detectable.":[245],"However,":[246],"it":[247],"still":[249],"rescue":[252],"these":[257],"transfected":[258],"reselection":[261],"suggesting":[262],"presence":[264],"containing":[268],"plasmid":[269],"portion":[272],"cells.":[275],"Analysis":[276],"DNA":[278],"indicated":[279],"that":[280],"vector":[283],"copy":[284],"number":[285],"reduced":[287],"30":[289],"copies":[290,299],"per":[291,300],"cell":[292,301],"selection,":[295],"approximately":[297],"0.3":[298],"20":[303],"hygromycin":[306],"B.":[307]},"counts_by_year":[{"year":2015,"cited_by_count":1},{"year":2014,"cited_by_count":1},{"year":2012,"cited_by_count":1}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2016-06-24T00:00:00"}