{"id":"https://openalex.org/W1979746830","doi":"https://doi.org/10.1089/hum.1997.8.6-659","title":"Recombinant Adeno-Associated Viral Vectors Mediate Long-Term Transgene Expression in Muscle","display_name":"Recombinant Adeno-Associated Viral Vectors Mediate Long-Term Transgene Expression in Muscle","publication_year":1997,"publication_date":"1997-04-10","ids":{"openalex":"https://openalex.org/W1979746830","doi":"https://doi.org/10.1089/hum.1997.8.6-659","mag":"1979746830","pmid":"https://pubmed.ncbi.nlm.nih.gov/9113506"},"language":"en","primary_location":{"id":"doi:10.1089/hum.1997.8.6-659","doi":"https://doi.org/10.1089/hum.1997.8.6-659","is_oa":false,"landing_page_url":"https://doi.org/10.1089/hum.1997.8.6-659","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/A5078328345","display_name":"Kelly Reed Clark","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"K. Reed Clark","raw_affiliation_strings":["Children's Hospital Research Foundation, Columbus, OH, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital Research Foundation, Columbus, OH, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5080838490","display_name":"Thomas J. Sferra","orcid":"https://orcid.org/0000-0001-6893-9880","observed_orcids":["https://orcid.org/0000-0001-6893-9880"]},"institutions":[{"id":"https://openalex.org/I1314135232","display_name":"Nationwide Children's Hospital","ror":"https://ror.org/003rfsp33","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1314135232"]},{"id":"https://openalex.org/I52357470","display_name":"The Ohio State University","ror":"https://ror.org/00rs6vg23","country_code":"US","type":"education","lineage":["https://openalex.org/I52357470"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Thomas J. Sferra","raw_affiliation_strings":["Children's Hospital Columbus","The Ohio State University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Children's Hospital Columbus","institution_ids":["https://openalex.org/I1314135232"]},{"raw_affiliation_string":"The Ohio State University","institution_ids":["https://openalex.org/I52357470"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5016866868","display_name":"Philip R. Johnson","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1314135232","display_name":"Nationwide Children's Hospital","ror":"https://ror.org/003rfsp33","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1314135232"]},{"id":"https://openalex.org/I52357470","display_name":"The Ohio State University","ror":"https://ror.org/00rs6vg23","country_code":"US","type":"education","lineage":["https://openalex.org/I52357470"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Philip R. Johnson","raw_affiliation_strings":["Address reprint requests to: Dr. Philip R. Johnson, Division of Molecular Medicine, Children's Hospital, Room W309, 700 Children's Drive, Columbus, OH 43205","Children's Hospital Columbus","The Ohio State University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Address reprint requests to: Dr. Philip R. Johnson, Division of Molecular Medicine, Children's Hospital, Room W309, 700 Children's Drive, Columbus, OH 43205","institution_ids":["https://openalex.org/I1314135232"]},{"raw_affiliation_string":"Children's Hospital Columbus","institution_ids":["https://openalex.org/I1314135232"]},{"raw_affiliation_string":"The Ohio State University","institution_ids":["https://openalex.org/I52357470"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5016866868"],"corresponding_institution_ids":["https://openalex.org/I1314135232","https://openalex.org/I52357470"],"apc_list":null,"apc_paid":null,"fwci":11.6894,"has_fulltext":false,"cited_by_count":150,"citation_normalized_percentile":{"value":0.99307297,"is_in_top_1_percent":true,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":89,"max":98},"biblio":{"volume":"8","issue":"6","first_page":"659","last_page":"669"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9990000128746033,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9990000128746033,"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":0.0005000000237487257,"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/T12400","display_name":"Neurogenetic and Muscular Disorders Research","score":0.00019999999494757503,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/recombinant-adeno-associated-virus","display_name":"recombinant adeno-associated virus","score":0.9079999923706055},{"id":"https://openalex.org/keywords/skeletal-muscle-transduction","display_name":"skeletal muscle transduction","score":0.8650000095367432},{"id":"https://openalex.org/keywords/long-term-transgene-expression","display_name":"long-term transgene expression","score":0.9229999780654907},{"id":"https://openalex.org/keywords/concatemeric-dna","display_name":"concatemeric DNA","score":0.5170000195503235},{"id":"https://openalex.org/keywords/immune-evasion","display_name":"immune evasion","score":0.5049999952316284},{"id":"https://openalex.org/keywords/neutralizing-antibodies","display_name":"neutralizing antibodies","score":0.9909999966621399},{"id":"https://openalex.org/keywords/vector-immunogenicity","display_name":"vector immunogenicity","score":0.5350000262260437},{"id":"https://openalex.org/keywords/inherited-myopathies","display_name":"inherited myopathies","score":0.7210000157356262},{"id":"https://openalex.org/keywords/lysosomal-storage-disorders","display_name":"lysosomal storage disease","score":0.9419999718666077}],"concepts":[{"id":"https://openalex.org/C102230213","wikidata":"https://www.wikidata.org/wiki/Q430117","display_name":"Transgene","level":3,"score":0.8327720165252686},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.7755535244941711},{"id":"https://openalex.org/C2778107364","wikidata":"https://www.wikidata.org/wiki/Q355989","display_name":"Adeno-associated virus","level":5,"score":0.5683000087738037},{"id":"https://openalex.org/C61797465","wikidata":"https://www.wikidata.org/wiki/Q1188986","display_name":"Term (time)","level":2,"score":0.5546862483024597},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5290573835372925},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.4880165159702301},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.44000911712646484},{"id":"https://openalex.org/C39060996","wikidata":"https://www.wikidata.org/wiki/Q7302706","display_name":"Recombinant virus","level":4,"score":0.43085137009620667},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.3832458555698395},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.3794223964214325},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.26501190662384033},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.2561552822589874},{"id":"https://openalex.org/C121332964","wikidata":"https://www.wikidata.org/wiki/Q413","display_name":"Physics","level":0,"score":0.13933023810386658},{"id":"https://openalex.org/C62520636","wikidata":"https://www.wikidata.org/wiki/Q944","display_name":"Quantum mechanics","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000229","descriptor_name":"Dependovirus","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D001616","descriptor_name":"beta-Galactosidase","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D004274","descriptor_name":"DNA, Recombinant","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":"D006367","descriptor_name":"HeLa Cells","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":"D008807","descriptor_name":"Mice, Inbred BALB C","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D018482","descriptor_name":"Muscle, Skeletal","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":true},{"descriptor_ui":"D019076","descriptor_name":"Transgenes","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"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.1997.8.6-659","doi":"https://doi.org/10.1089/hum.1997.8.6-659","is_oa":false,"landing_page_url":"https://doi.org/10.1089/hum.1997.8.6-659","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:9113506","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/9113506","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":"Journal Article"}],"best_oa_location":null,"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.9537000060081482}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":62,"referenced_works":["https://openalex.org/W32161039","https://openalex.org/W37310352","https://openalex.org/W43014397","https://openalex.org/W114318986","https://openalex.org/W1490724066","https://openalex.org/W1519383882","https://openalex.org/W1645009826","https://openalex.org/W1795219398","https://openalex.org/W1807225431","https://openalex.org/W1879335341","https://openalex.org/W1929630117","https://openalex.org/W1964914116","https://openalex.org/W1965172479","https://openalex.org/W1967937424","https://openalex.org/W1970459261","https://openalex.org/W1970708694","https://openalex.org/W1973754802","https://openalex.org/W1983903557","https://openalex.org/W1990748900","https://openalex.org/W2009415155","https://openalex.org/W2009761723","https://openalex.org/W2014010760","https://openalex.org/W2015219383","https://openalex.org/W2016390889","https://openalex.org/W2016782363","https://openalex.org/W2019612944","https://openalex.org/W2021873030","https://openalex.org/W2025728946","https://openalex.org/W2026296977","https://openalex.org/W2029426142","https://openalex.org/W2031598154","https://openalex.org/W2036650398","https://openalex.org/W2038036971","https://openalex.org/W2051729665","https://openalex.org/W2061275081","https://openalex.org/W2067587032","https://openalex.org/W2068065419","https://openalex.org/W2070382042","https://openalex.org/W2077224588","https://openalex.org/W2079209506","https://openalex.org/W2082275088","https://openalex.org/W2084017165","https://openalex.org/W2084210336","https://openalex.org/W2085778900","https://openalex.org/W2097072158","https://openalex.org/W2131095976","https://openalex.org/W2139784464","https://openalex.org/W2151902657","https://openalex.org/W2159706578","https://openalex.org/W2245959075","https://openalex.org/W2273251931","https://openalex.org/W2273876198","https://openalex.org/W2326865483","https://openalex.org/W2336594850","https://openalex.org/W2401007665","https://openalex.org/W2403288307","https://openalex.org/W2409119244","https://openalex.org/W2410236485","https://openalex.org/W2412570611","https://openalex.org/W2470430297","https://openalex.org/W4240798725","https://openalex.org/W4323238558"],"related_works":["https://openalex.org/W1974877896","https://openalex.org/W1519383882","https://openalex.org/W2007854533","https://openalex.org/W2410404480","https://openalex.org/W2044286471","https://openalex.org/W2335611176","https://openalex.org/W2145578753","https://openalex.org/W2368889144","https://openalex.org/W1512204385","https://openalex.org/W1552712529"],"abstract_inverted_index":{"Gene":[0],"transfer":[1,43,141],"to":[2,44,103,109,142,153,187,205,211,223],"muscle":[3,22,45,157],"holds":[4,133],"overt":[5],"promise":[6,135],"for":[7,27,139,169,220],"the":[8,50,53,58,180],"treatment":[9],"of":[10,29,52,61,192,195],"inherited":[11],"myopathies,":[12],"lysosomal":[13],"storage":[14],"disorders,":[15],"and":[16,57,90,127,132,167],"serum":[17],"protein":[18],"deficiencies.":[19],"In":[20,64],"addition,":[21],"could":[23],"provide":[24],"a":[25,70,120,137,189,201],"reservoir":[26],"delivery":[28,55,130,222],"therapeutic":[30],"molecules":[31],"like":[32],"blood":[33],"clotting":[34],"factors,":[35],"erythropoietin,":[36],"or":[37],"insulin.":[38],"To":[39],"date,":[40],"successful":[41],"gene":[42,62,129,140,221],"has":[46],"been":[47],"limited":[48],"by":[49,179],"inefficiency":[51],"vector":[54,71,100,138,150,196],"systems":[56],"transient":[59],"nature":[60],"expression.":[63],"this":[65],"paper,":[66],"we":[67],"show":[68],"that":[69,116],"based":[72],"on":[73],"recombinant":[74,146],"adeno-associated":[75,147],"virus":[76,148],"(rAAV)":[77,149],"can":[78],"efficiently":[79],"transduce":[80,154],"adult":[81],"mouse":[82,155],"skeletal":[83,156],"muscle.":[84,144,224],"Transduced":[85,174],"myofibers":[86],"escape":[87],"immune":[88,181],"elimination":[89],"transgene":[91],"expression":[92,160],"is":[93],"robust":[94],"beyond":[95],"5":[96,172],"months.":[97,173],"Importantly,":[98],"input":[99],"DNA":[101,197],"appears":[102],"undergo":[104],"conversion":[105],"from":[106,203],"single-stranded":[107],"genomes":[108],"high-molecular-weight":[110,206],"concatameric":[111],"forms.":[112,207],"These":[113],"data":[114],"suggest":[115],"rAAV":[117,208],"might":[118],"have":[119,212],"significant":[121,134,213],"advantage":[122],"over":[123,215],"many":[124],"other":[125,216],"viral":[126],"nonviral":[128],"methods,":[131],"as":[136],"mature":[143],"A":[145],"was":[151,162],"shown":[152],"effectively.":[158],"Transgene":[159],"(\u03b2-galactosidase)":[161],"evident":[163],"early":[164],"after":[165,198],"injection":[166,199],"lasted":[168],"more":[170],"than":[171],"cells":[175],"were":[176],"not":[177],"eliminated":[178],"system,":[182],"but":[183],"neutralizing":[184],"antibodies":[185],"appeared":[186],"block":[188],"second":[190],"dose":[191],"vector.":[193],"Analysis":[194],"showed":[200],"shift":[202],"low-":[204],"vectors":[209],"appear":[210],"advantages":[214],"currently":[217],"available":[218],"methods":[219]},"counts_by_year":[{"year":2026,"cited_by_count":1},{"year":2024,"cited_by_count":1},{"year":2020,"cited_by_count":3},{"year":2019,"cited_by_count":3},{"year":2017,"cited_by_count":2},{"year":2016,"cited_by_count":2},{"year":2015,"cited_by_count":5},{"year":2014,"cited_by_count":3},{"year":2013,"cited_by_count":4}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2016-06-24T00:00:00"}
