{"id":"https://openalex.org/W43014397","doi":null,"title":"An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction.","display_name":"An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction.","publication_year":1995,"publication_date":"1995-01-01","ids":{"openalex":"https://openalex.org/W43014397","mag":"43014397","pmid":"https://pubmed.ncbi.nlm.nih.gov/7712331"},"language":"en","primary_location":{"id":"pmid:7712331","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/7712331","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":"Comparative Study"},"volume":null,"type":"article","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/A5010005871","display_name":"Terence R. Flotte","orcid":"https://orcid.org/0000-0002-8255-0588","observed_orcids":["https://orcid.org/0000-0002-8255-0588"]},"institutions":[{"id":"https://openalex.org/I145311948","display_name":"Johns Hopkins University","ror":"https://ror.org/00za53h95","country_code":"US","type":"education","lineage":["https://openalex.org/I145311948"]},{"id":"https://openalex.org/I2799853436","display_name":"Johns Hopkins Medicine","ror":"https://ror.org/037zgn354","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I2799853436"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"T R Flotte","raw_affiliation_strings":["Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21287-2533"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21287-2533","institution_ids":["https://openalex.org/I145311948","https://openalex.org/I2799853436"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5036240273","display_name":"X Barraza-Ortiz","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"X Barraza-Ortiz","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5022038622","display_name":"Rikki Solow","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"R Solow","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5028991111","display_name":"Sandra A. Afione","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"S A Afione","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111820586","display_name":"Barbara J. Carter","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"B J Carter","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"last","author":{"id":"https://openalex.org/A5029220053","display_name":"William B. Guggino","orcid":"https://orcid.org/0000-0002-2153-9764","observed_orcids":["https://orcid.org/0000-0002-2153-9764"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"W B Guggino","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":9.9813,"has_fulltext":false,"cited_by_count":100,"citation_normalized_percentile":{"value":0.98660909,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":89,"max":97},"biblio":{"volume":"2","issue":"1","first_page":"29","last_page":"37"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":1.0,"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":1.0,"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":9.999999747378752e-05,"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/T11663","display_name":"Viral Infectious Diseases and Gene Expression in Insects","score":0.0,"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/adeno-associated-virus-vectors","display_name":"adeno-associated virus vectors","score":0.9559999704360962},{"id":"https://openalex.org/keywords/in-vivo-transduction","display_name":"in vivo transduction","score":0.6899999976158142},{"id":"https://openalex.org/keywords/lacz-reporter","display_name":"lacZ reporter","score":0.8169999718666077},{"id":"https://openalex.org/keywords/systemic-gene-therapy","display_name":"systemic gene therapy","score":0.5759999752044678},{"id":"https://openalex.org/keywords/airway-epithelium","display_name":"airway epithelium","score":0.8399999737739563},{"id":"https://openalex.org/keywords/spleen","display_name":"spleen","score":0.593999981880188}],"concepts":[{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.8034677505493164},{"id":"https://openalex.org/C15152581","wikidata":"https://www.wikidata.org/wiki/Q7833966","display_name":"Transduction (biophysics)","level":2,"score":0.7715772390365601},{"id":"https://openalex.org/C2778107364","wikidata":"https://www.wikidata.org/wiki/Q355989","display_name":"Adeno-associated virus","level":5,"score":0.7244364023208618},{"id":"https://openalex.org/C161733203","wikidata":"https://www.wikidata.org/wiki/Q911828","display_name":"Reporter gene","level":4,"score":0.7056077122688293},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.6334824562072754},{"id":"https://openalex.org/C54009773","wikidata":"https://www.wikidata.org/wiki/Q1429031","display_name":"Transfection","level":3,"score":0.6180713176727295},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.5971131324768066},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.5595723390579224},{"id":"https://openalex.org/C81885089","wikidata":"https://www.wikidata.org/wiki/Q189082","display_name":"Cell culture","level":2,"score":0.5475127696990967},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.5211789011955261},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.5063725709915161},{"id":"https://openalex.org/C135983454","wikidata":"https://www.wikidata.org/wiki/Q2790163","display_name":"Gene delivery","level":4,"score":0.4929521083831787},{"id":"https://openalex.org/C2522874641","wikidata":"https://www.wikidata.org/wiki/Q808","display_name":"Virus","level":2,"score":0.4861166477203369},{"id":"https://openalex.org/C174510640","wikidata":"https://www.wikidata.org/wiki/Q489618","display_name":"HEK 293 cells","level":3,"score":0.4753453731536865},{"id":"https://openalex.org/C22744801","wikidata":"https://www.wikidata.org/wiki/Q172778","display_name":"Plasmid","level":3,"score":0.45462751388549805},{"id":"https://openalex.org/C207001950","wikidata":"https://www.wikidata.org/wiki/Q141124","display_name":"In vivo","level":2,"score":0.45348426699638367},{"id":"https://openalex.org/C39060996","wikidata":"https://www.wikidata.org/wiki/Q7302706","display_name":"Recombinant virus","level":4,"score":0.4269426167011261},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.4004846513271332},{"id":"https://openalex.org/C150194340","wikidata":"https://www.wikidata.org/wiki/Q26972","display_name":"Gene expression","level":3,"score":0.3929577171802521},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.20979219675064087},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000229","descriptor_name":"Dependovirus","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002460","descriptor_name":"Cell Line","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004268","descriptor_name":"DNA-Binding Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D004274","descriptor_name":"DNA, Recombinant","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D007763","descriptor_name":"Lac Operon","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008168","descriptor_name":"Lung","qualifier_ui":"Q000201","qualifier_name":"enzymology","is_major_topic":false},{"descriptor_ui":"D008168","descriptor_name":"Lung","qualifier_ui":"Q000821","qualifier_name":"virology","is_major_topic":false},{"descriptor_ui":"D008810","descriptor_name":"Mice, Inbred C57BL","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D011401","descriptor_name":"Promoter Regions, Genetic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D013154","descriptor_name":"Spleen","qualifier_ui":"Q000201","qualifier_name":"enzymology","is_major_topic":false},{"descriptor_ui":"D013154","descriptor_name":"Spleen","qualifier_ui":"Q000821","qualifier_name":"virology","is_major_topic":false},{"descriptor_ui":"D014162","descriptor_name":"Transfection","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D014764","descriptor_name":"Viral Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D015870","descriptor_name":"Gene Expression","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016325","descriptor_name":"HIV Long Terminal Repeat","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":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":2,"locations":[{"id":"pmid:7712331","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/7712331","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":"Comparative Study"},{"id":"mag:43014397","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/7712331","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","score":0.9763000011444092,"display_name":"Good health and well-being"}],"sustainable_development_goals_aurora":[],"study_designs":[],"awards":[{"id":"https://openalex.org/G3092487464","display_name":"Gene and Pharmacological Therapies for Cystic Fibrosis","funder_award_id":"P01HL051811","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G8269929776","display_name":"CFTR/Regulation of CL Secretion in Normal and CF Airways","funder_award_id":"R01HL047122","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"}],"funders":[{"id":"https://openalex.org/F4320337338","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W2076136811","https://openalex.org/W7096289724","https://openalex.org/W2035249958","https://openalex.org/W1983903557","https://openalex.org/W2063854877","https://openalex.org/W2145775145","https://openalex.org/W2323927506","https://openalex.org/W2075808672","https://openalex.org/W2099350945","https://openalex.org/W4223596074"],"abstract_inverted_index":{"Adeno-associated":[0],"virus":[1,92],"(AAV)":[2],"vectors":[3,21],"are":[4],"potentially":[5],"useful":[6],"for":[7,191],"gene":[8,46,168,192],"therapy":[9],"of":[10,13,19,28,44,56,63,86,104,113,132,138,164,171,183],"a":[11,101,129],"number":[12],"human":[14,90],"diseases.":[15],"However,":[16],"the":[17,26,49,54,89,111,135,139,165,172,179],"use":[18],"these":[20,75,184],"has":[22],"been":[23],"limited":[24],"by":[25,117],"lack":[27],"stable":[29],"vector-packaging":[30],"cell":[31,38,65,119],"lines.":[32,66],"The":[33,145],"difficulties":[34],"in":[35,100,134,169,180],"developing":[36],"packaging":[37,105,136],"lines":[39],"relate":[40],"to":[41,53,59,77,155],"low":[42],"levels":[43],"rep":[45,87],"expression":[47,85,163],"from":[48,88],"AAV-p5":[50],"promoter,":[51],"and":[52,141,160,175,187],"propensity":[55],"Rep":[57],"proteins":[58],"suppress":[60],"continued":[61],"growth":[62],"immortalized":[64],"We":[67],"describe":[68],"here":[69],"two":[70],"new":[71],"techniques":[72],"which":[73],"allow":[74],"problems":[76],"be":[78],"circumvented.":[79],"First,":[80],"we":[81,108],"have":[82,109],"demonstrated":[83],"that":[84],"immunodeficiency":[91],"(HIV)":[93],"long":[94],"terminal":[95],"repeat":[96],"(LTR)":[97],"promoter":[98],"results":[99],"10-fold":[102],"improvement":[103],"efficiency.":[106],"Second,":[107],"overcome":[110],"inefficiency":[112],"vector":[114,147,185],"plasmid":[115],"transfection":[116],"generating":[118],"populations":[120],"containing":[121],"rescuable":[122],"AAV":[123],"recombinant":[124,143],"genomes.":[125],"These":[126],"improvements":[127],"yielded":[128],"net":[130],"increase":[131],"50-fold":[133],"efficiency":[137],"AAVp5neo":[140],"AAVp5lacZ":[142,146],"vectors.":[144],"packaged":[148],"with":[149],"this":[150],"method":[151],"was":[152],"administered":[153],"systemically":[154],"recently":[156],"weaned":[157],"C57BL":[158],"mice,":[159],"mediated":[161],"efficient":[162],"beta-galactosidase":[166],"reporter":[167],"cells":[170],"airway":[173],"epithelium":[174],"spleen.":[176],"This":[177],"indicates":[178],"vivo":[181],"activity":[182],"stocks,":[186],"their":[188],"potential":[189],"utility":[190],"therapy.":[193]},"counts_by_year":[{"year":2018,"cited_by_count":3},{"year":2017,"cited_by_count":2},{"year":2014,"cited_by_count":3},{"year":2013,"cited_by_count":1}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2016-06-24T00:00:00"}
