{"id":"https://openalex.org/W2036921663","doi":"https://doi.org/10.1089/hum.1994.5.9-1121","title":"Increasing Expression of the Normal Human CFTR cDNA in Cystic Fibrosis Epithelial Cells Results in a Progressive Increase in the Level of CFTR Protein Expression, but a Limit on the Level of cAMP-Stimulated Chloride Secretion","display_name":"Increasing Expression of the Normal Human CFTR cDNA in Cystic Fibrosis Epithelial Cells Results in a Progressive Increase in the Level of CFTR Protein Expression, but a Limit on the Level of cAMP-Stimulated Chloride Secretion","publication_year":1994,"publication_date":"1994-09-01","ids":{"openalex":"https://openalex.org/W2036921663","doi":"https://doi.org/10.1089/hum.1994.5.9-1121","mag":"2036921663","pmid":"https://pubmed.ncbi.nlm.nih.gov/7530494"},"language":"en","primary_location":{"id":"doi:10.1089/hum.1994.5.9-1121","doi":"https://doi.org/10.1089/hum.1994.5.9-1121","is_oa":false,"landing_page_url":"https://doi.org/10.1089/hum.1994.5.9-1121","pdf_url":null,"source":{"id":"https://openalex.org/S39730559","display_name":"Human Gene Therapy","issn_l":"1043-0342","issn":["1043-0342","1557-7422"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320443","host_organization_name":"Mary Ann Liebert, Inc.","host_organization_lineage":["https://openalex.org/P4310320443","https://openalex.org/P4310320017"],"host_organization_lineage_names":["Mary Ann Liebert, Inc.","SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Human Gene Therapy","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"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/A5081230131","display_name":"Melissa A. Rosenfeld","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1299303238","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88","country_code":"US","type":"government","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238"]},{"id":"https://openalex.org/I4210106489","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26","country_code":"US","type":"facility","lineage":["https://openalex.org/I1299022934","https://openalex.org/I1299303238","https://openalex.org/I4210106489"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Melissa A. Rosenfeld","raw_affiliation_strings":["Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892","institution_ids":["https://openalex.org/I1299303238","https://openalex.org/I4210106489"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5062424124","display_name":"Stephen J. Rosenfeld","orcid":"https://orcid.org/0000-0002-2886-0227","observed_orcids":["https://orcid.org/0000-0002-2886-0227"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Stephen J. Rosenfeld","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5030654528","display_name":"Claire J. Danel","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Claire Danel","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111563489","display_name":"Tyrone C. Banks","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Tyrone C. Banks","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"last","author":{"id":"https://openalex.org/A5064433832","display_name":"Ronald G. Crystal","orcid":"https://orcid.org/0000-0001-6177-6839","observed_orcids":["https://orcid.org/0000-0001-6177-6839"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Ronald G. Crystal","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5081230131"],"corresponding_institution_ids":["https://openalex.org/I1299303238","https://openalex.org/I4210106489"],"apc_list":null,"apc_paid":null,"fwci":2.3333,"has_fulltext":false,"cited_by_count":14,"citation_normalized_percentile":{"value":0.88200109,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":88,"max":93},"biblio":{"volume":"5","issue":"9","first_page":"1121","last_page":"1129"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10665","display_name":"Cystic Fibrosis Research Advances","score":0.9761000275611877,"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":0.9761000275611877,"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":0.021199999377131462,"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":0.00019999999494757503,"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":0.9990000128746033},{"id":"https://openalex.org/keywords/cftr","display_name":"CFTR","score":0.9229999780654907},{"id":"https://openalex.org/keywords/adenovirus-vectors","display_name":"adenoviral vector","score":0.7889999747276306},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7799999713897705},{"id":"https://openalex.org/keywords/cfpac-1-cells","display_name":"CFPAC-1 cells","score":0.8579999804496765},{"id":"https://openalex.org/keywords/pancreatic-epithelial-cells","display_name":"pancreatic epithelial cells","score":0.9409999847412109},{"id":"https://openalex.org/keywords/chloride-efflux","display_name":"chloride efflux","score":0.7879999876022339},{"id":"https://openalex.org/keywords/multiplicity-of-infection","display_name":"multiplicity of infection","score":0.8429999947547913}],"concepts":[{"id":"https://openalex.org/C2778428886","wikidata":"https://www.wikidata.org/wiki/Q420470","display_name":"Cystic fibrosis transmembrane conductance regulator","level":3,"score":0.8760287165641785},{"id":"https://openalex.org/C2776938444","wikidata":"https://www.wikidata.org/wiki/Q178194","display_name":"Cystic fibrosis","level":2,"score":0.8652603626251221},{"id":"https://openalex.org/C203233990","wikidata":"https://www.wikidata.org/wiki/Q1511135","display_name":"Multiplicity of infection","level":3,"score":0.7232545018196106},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.5403341054916382},{"id":"https://openalex.org/C187882448","wikidata":"https://www.wikidata.org/wiki/Q283478","display_name":"Complementary DNA","level":3,"score":0.5013976097106934},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.4929203987121582},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.4881215989589691},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.48574739694595337},{"id":"https://openalex.org/C150194340","wikidata":"https://www.wikidata.org/wiki/Q26972","display_name":"Gene expression","level":3,"score":0.47674664855003357},{"id":"https://openalex.org/C200082930","wikidata":"https://www.wikidata.org/wiki/Q22327148","display_name":"Efflux","level":2,"score":0.4548807740211487},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.44526100158691406},{"id":"https://openalex.org/C105580179","wikidata":"https://www.wikidata.org/wiki/Q188928","display_name":"Messenger RNA","level":3,"score":0.4344020187854767},{"id":"https://openalex.org/C54009773","wikidata":"https://www.wikidata.org/wiki/Q1429031","display_name":"Transfection","level":3,"score":0.4295055568218231},{"id":"https://openalex.org/C202751555","wikidata":"https://www.wikidata.org/wiki/Q221681","display_name":"In vitro","level":2,"score":0.32956385612487793},{"id":"https://openalex.org/C185592680","wikidata":"https://www.wikidata.org/wiki/Q2329","display_name":"Chemistry","level":0,"score":0.32912635803222656},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3211491107940674},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.12811869382858276},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.07743275165557861}],"mesh":[{"descriptor_ui":"D000230","descriptor_name":"Adenocarcinoma","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":false},{"descriptor_ui":"D000230","descriptor_name":"Adenocarcinoma","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D000242","descriptor_name":"Cyclic AMP","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":true},{"descriptor_ui":"D000260","descriptor_name":"Adenoviruses, Human","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","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":"D003110","descriptor_name":"Colonic Neoplasms","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D003550","descriptor_name":"Cystic Fibrosis","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":false},{"descriptor_ui":"D003550","descriptor_name":"Cystic Fibrosis","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":true},{"descriptor_ui":"D004848","descriptor_name":"Epithelium","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D004848","descriptor_name":"Epithelium","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D005576","descriptor_name":"Colforsin","qualifier_ui":"Q000494","qualifier_name":"pharmacology","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":"D008325","descriptor_name":"Mammary Neoplasms, Experimental","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D008565","descriptor_name":"Membrane Proteins","qualifier_ui":"Q000096","qualifier_name":"biosynthesis","is_major_topic":true},{"descriptor_ui":"D008565","descriptor_name":"Membrane Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D010190","descriptor_name":"Pancreatic Neoplasms","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":false},{"descriptor_ui":"D010190","descriptor_name":"Pancreatic Neoplasms","qualifier_ui":"Q000473","qualifier_name":"pathology","is_major_topic":false},{"descriptor_ui":"D011993","descriptor_name":"Recombinant Fusion Proteins","qualifier_ui":"Q000096","qualifier_name":"biosynthesis","is_major_topic":true},{"descriptor_ui":"D011993","descriptor_name":"Recombinant Fusion Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D012636","descriptor_name":"Secretory Rate","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D014407","descriptor_name":"Tumor Cells, Cultured","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018076","descriptor_name":"DNA, Complementary","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D019005","descriptor_name":"Cystic Fibrosis Transmembrane Conductance Regulator","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":2,"locations":[{"id":"doi:10.1089/hum.1994.5.9-1121","doi":"https://doi.org/10.1089/hum.1994.5.9-1121","is_oa":false,"landing_page_url":"https://doi.org/10.1089/hum.1994.5.9-1121","pdf_url":null,"source":{"id":"https://openalex.org/S39730559","display_name":"Human Gene Therapy","issn_l":"1043-0342","issn":["1043-0342","1557-7422"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320443","host_organization_name":"Mary Ann Liebert, Inc.","host_organization_lineage":["https://openalex.org/P4310320443","https://openalex.org/P4310320017"],"host_organization_lineage_names":["Mary Ann Liebert, Inc.","SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Human Gene Therapy","raw_type":"journal-article"},{"id":"pmid:7530494","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/7530494","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":"Human gene therapy","raw_type":"Comparative Study"}],"best_oa_location":null,"sustainable_development_goals":[{"score":0.9789000153541565,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":30,"referenced_works":["https://openalex.org/W1516696899","https://openalex.org/W1786021994","https://openalex.org/W1959459432","https://openalex.org/W1965372133","https://openalex.org/W1969697265","https://openalex.org/W1975971473","https://openalex.org/W1980401440","https://openalex.org/W1981015157","https://openalex.org/W1993935349","https://openalex.org/W1995240556","https://openalex.org/W2016532191","https://openalex.org/W2020301682","https://openalex.org/W2035714544","https://openalex.org/W2039599894","https://openalex.org/W2044296969","https://openalex.org/W2052588826","https://openalex.org/W2056262259","https://openalex.org/W2060333964","https://openalex.org/W2060917321","https://openalex.org/W2079234445","https://openalex.org/W2084491822","https://openalex.org/W2124314287","https://openalex.org/W2125391335","https://openalex.org/W2129859001","https://openalex.org/W2141365582","https://openalex.org/W2153450253","https://openalex.org/W2168225085","https://openalex.org/W2178159662","https://openalex.org/W2210537951","https://openalex.org/W3145894777"],"related_works":["https://openalex.org/W2037872751","https://openalex.org/W1981015157","https://openalex.org/W2041549282","https://openalex.org/W2409683519","https://openalex.org/W2433844444","https://openalex.org/W2079791074","https://openalex.org/W2085490249","https://openalex.org/W262255493","https://openalex.org/W1991604425","https://openalex.org/W1978287455"],"abstract_inverted_index":{"Cystic":[0],"fibrosis":[1],"(CF)":[2],"results":[3],"from":[4,173],"mutations":[5],"of":[6,19,33,63,103,130,154,170,190,197,210,220,243,259,291,306],"the":[7,15,48,61,91,109,125,155,188,208,218,244,251],"CF":[8,67,92,213],"transmembrane":[9],"conductance":[10],"regulator":[11],"(CFTR)":[12],"gene":[13,68,183,214],"and":[14,40,113,118,121,136,263],"consequent":[16],"defective":[17],"regulation":[18],"cAMP-stimulated":[20,85,146,191,233,272,300],"Cl\u2013":[21,86,192,234,301],"permeability":[22],"across":[23],"epithelial":[24,94,185,297],"cell":[25,95],"apical":[26],"membranes.":[27],"Given":[28],"that":[29,41,180,296],"in":[30,53,60,164,207,275,286],"vitro":[31],"transfer":[32,47],"normal":[34,49,110,252],"CFTR":[35,51,74,111,116,122,134,142,165,198,222,254,261,267,307],"cDNA":[36,52],"corrects":[37],"this":[38],"defect":[39],"recombinant":[42,106,245],"adenovirus":[43],"(Ad)":[44,201],"vectors":[45,56,79,202,227],"can":[46],"human":[50,253],"vivo,":[54],"Ad":[55,78,107,226,246],"have":[57,81,204,229],"significant":[58,205],"potential":[59,206],"development":[62,209],"effective":[64],"strategies":[65,211],"for":[66,115,212],"therapy.":[69,215],"One":[70],"concern":[71],"is":[72],"whether":[73,221],"overexpression":[75,223],"achievable":[76,224],"with":[77,100,151,167,225,240,278,288],"may":[80,203],"untoward":[82,230],"effects":[83,231],"on":[84,232],"efflux.":[87],"To":[88,216],"address":[89,217],"this,":[90],"pancreatic":[93],"line":[96],"CFPAC-1":[97,236],"was":[98,149,160,269,282],"infected":[99,239,277],"increasing":[101,168,195,241,257,289,304],"doses":[102,153,169,242,258,290],"AdCFTR,":[104,248,260,279],"a":[105],"containing":[108],"cDNA,":[112],"analyzed":[114],"mRNA":[117,135,262],"protein":[119,137,264],"levels":[120,138,196,265,305],"function.":[123],"As":[124],"AdCFTR":[126,171],"dose":[127],"increased":[128],"[multiplicity":[129],"infection":[131],"(moi)":[132],"0\u20131,000],":[133],"increased.":[139,266],"However,":[140],"while":[141],"function":[143,166,268,287],"measured":[144],"by":[145,271],"36Cl\u2013":[147,273],"efflux":[148,274],"observed":[150,270],"low":[152],"vector":[156],"(moi":[157,172],"20),":[158],"there":[159,281],"no":[161,283],"further":[162,284],"increase":[163,285],"20":[174],"to":[175],"1,000).":[176],"These":[177,293],"data":[178,294],"suggest":[179,295],"after":[181],"AdCFTR-mediated":[182],"transfer,":[184],"cells":[186,237,276,298],"limit":[187,299],"level":[189],"secretion":[193,302],"despite":[194,303],"protein.":[199,308],"Adenovirus":[200],"question":[219],"could":[228],"efflux,":[235],"were":[238],"vector,":[247],"which":[249],"contains":[250],"CDNA.":[255],"With":[256],"but":[280],"AdCFTR.":[292]},"counts_by_year":[{"year":2016,"cited_by_count":1}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2016-06-24T00:00:00"}