{"id":"https://openalex.org/W2138760162","doi":null,"title":"Targeted expression of two proteins in neural retina using self-inactivating, insulated lentiviral vectors carrying two internal independent promoters.","display_name":"Targeted expression of two proteins in neural retina using self-inactivating, insulated lentiviral vectors carrying two internal independent promoters.","publication_year":2007,"publication_date":"2007-10-18","ids":{"openalex":"https://openalex.org/W2138760162","mag":"2138760162","pmid":"https://pubmed.ncbi.nlm.nih.gov/17982424"},"language":"en","primary_location":{"id":"pmid:17982424","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/17982424","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 vision","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/A5082544318","display_name":"Susan L. Semple\u2010Rowland","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I33213144","display_name":"University of Florida","ror":"https://ror.org/02y3ad647","country_code":"US","type":"education","lineage":["https://openalex.org/I33213144"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Susan L Semple-Rowland","raw_affiliation_strings":["Department of Neuroscience, University of Florida McKnight Brain Institute, Gainesville, FL 32611, USA. rowland@mbi.ufl.edu"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neuroscience, University of Florida McKnight Brain Institute, Gainesville, FL 32611, USA. rowland@mbi.ufl.edu","institution_ids":["https://openalex.org/I33213144"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048656934","display_name":"Kristofer S Eccles","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Kristofer S Eccles","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"last","author":{"id":"https://openalex.org/A5019004888","display_name":"Elizabeth J Humberstone","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Elizabeth J Humberstone","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":1.6943,"has_fulltext":false,"cited_by_count":25,"citation_normalized_percentile":{"value":0.85022086,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":87,"max":97},"biblio":{"volume":"13","issue":null,"first_page":"2001","last_page":"11"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.8827999830245972,"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":0.8827999830245972,"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/T10691","display_name":"Retinal Development and Disorders","score":0.10729999840259552,"subfield":{"id":"https://openalex.org/subfields/2731","display_name":"Ophthalmology"},"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/T10725","display_name":"RNA Interference and Gene Delivery","score":0.00430000014603138,"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/self-inactivating-lentivirus","display_name":"self-inactivating lentivirus","score":0.7879999876022339},{"id":"https://openalex.org/keywords/gcap2","display_name":"GCAP2","score":0.7900000214576721},{"id":"https://openalex.org/keywords/interphotoreceptor-retinoid-binding-protein","display_name":"interphotoreceptor retinoid-binding protein","score":0.8569999933242798},{"id":"https://openalex.org/keywords/rhodopsin-promoter","display_name":"rhodopsin promoter","score":0.8299999833106995},{"id":"https://openalex.org/keywords/cone-photoreceptors","display_name":"cone photoreceptors","score":0.6179999709129333},{"id":"https://openalex.org/keywords/rod-photoreceptors","display_name":"rod photoreceptors","score":0.9810000061988831},{"id":"https://openalex.org/keywords/transgene-expression","display_name":"transgene expression","score":0.4359999895095825},{"id":"https://openalex.org/keywords/transduction-efficiency","display_name":"transduction efficiency","score":0.3269999921321869},{"id":"https://openalex.org/keywords/photoreceptor-degeneration","display_name":"photoreceptor degeneration","score":0.3580000102519989}],"concepts":[{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.7298099994659424},{"id":"https://openalex.org/C142613039","wikidata":"https://www.wikidata.org/wiki/Q272983","display_name":"Green fluorescent protein","level":3,"score":0.6679115295410156},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.5442535877227783},{"id":"https://openalex.org/C101762097","wikidata":"https://www.wikidata.org/wiki/Q224093","display_name":"Promoter","level":4,"score":0.5409787893295288},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.5363838076591492},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.4626278579235077},{"id":"https://openalex.org/C150194340","wikidata":"https://www.wikidata.org/wiki/Q26972","display_name":"Gene expression","level":3,"score":0.31477004289627075},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3083873391151428},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.18503600358963013},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.15050992369651794}],"mesh":[{"descriptor_ui":"D000099214","descriptor_name":"Retinol-Binding Proteins, Interstitial","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":"D002642","descriptor_name":"Chick Embryo","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005136","descriptor_name":"Eye Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D008164","descriptor_name":"Luminescent Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D011401","descriptor_name":"Promoter Regions, Genetic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D012160","descriptor_name":"Retina","qualifier_ui":"Q000196","qualifier_name":"embryology","is_major_topic":false},{"descriptor_ui":"D012160","descriptor_name":"Retina","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":true},{"descriptor_ui":"D012177","descriptor_name":"Retinol-Binding Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D012243","descriptor_name":"Rhodopsin","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D014018","descriptor_name":"Tissue Distribution","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D015870","descriptor_name":"Gene Expression","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016086","descriptor_name":"Lentivirus","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D016086","descriptor_name":"Lentivirus","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":false},{"descriptor_ui":"D018390","descriptor_name":"Gene Targeting","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D019076","descriptor_name":"Transgenes","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D020648","descriptor_name":"Peptide Elongation Factor 1","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D038301","descriptor_name":"Virus Inactivation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D049452","descriptor_name":"Green Fluorescent Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051604","descriptor_name":"Guanylate Cyclase-Activating Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false}],"locations_count":2,"locations":[{"id":"pmid:17982424","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/17982424","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 vision","raw_type":"Journal Article"},{"id":"mag:2138760162","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/17982424","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":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.8371999859809875}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1218626370","display_name":"Rescue of GUCY1*B Phenotype Using Somatic Gene Therapy","funder_award_id":"R01EY011388","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G5262378246","display_name":"VISION RESEARCH CORE","funder_award_id":"P30EY008571","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"}],"funders":[{"id":"https://openalex.org/F4320337350","display_name":"National Eye Institute","ror":"https://ror.org/03wkg3b53"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":32,"referenced_works":["https://openalex.org/W73310602","https://openalex.org/W1552714324","https://openalex.org/W1588415812","https://openalex.org/W1769569140","https://openalex.org/W1971723950","https://openalex.org/W1976365653","https://openalex.org/W1984003568","https://openalex.org/W1995004137","https://openalex.org/W1996875707","https://openalex.org/W2000853857","https://openalex.org/W2003858277","https://openalex.org/W2010303814","https://openalex.org/W2022682222","https://openalex.org/W2029782611","https://openalex.org/W2031062005","https://openalex.org/W2045383744","https://openalex.org/W2049466728","https://openalex.org/W2058005281","https://openalex.org/W2072440756","https://openalex.org/W2079020236","https://openalex.org/W2087264985","https://openalex.org/W2087303190","https://openalex.org/W2087690559","https://openalex.org/W2094595339","https://openalex.org/W2094988427","https://openalex.org/W2118846517","https://openalex.org/W2141354154","https://openalex.org/W2181781202","https://openalex.org/W2318615532","https://openalex.org/W2320400608","https://openalex.org/W2350853133","https://openalex.org/W2411465214"],"related_works":["https://openalex.org/W2129262428","https://openalex.org/W1173194423","https://openalex.org/W2160294802","https://openalex.org/W2073030837","https://openalex.org/W2021625998","https://openalex.org/W4240890411","https://openalex.org/W2094988427","https://openalex.org/W2008512248","https://openalex.org/W1835972708","https://openalex.org/W2154997400"],"abstract_inverted_index":{"PURPOSE:":[0],"There":[1],"is":[2,60,409,478],"increasing":[3],"interest":[4],"in":[5,57,77,98,102,206,224,269,302,309,322,330,344,378,389,393,422,491,497],"developing":[6,168],"viral":[7],"vectors":[8,22,39,59,82,109,260,416,430,439],"capable":[9,479],"of":[10,54,66,74,167,201,211,228,234,249,265,278,282,290,403,444,457,480,483,502],"reliably":[11,62,94,418],"delivering":[12],"multiple":[13,484,489],"therapeutic":[14],"genes":[15,485],"to":[16,40,70,217,238,411,447,460,467,472,486,506],"targeted":[17,99],"cell":[18],"populations.":[19],"Currently,":[20],"bicistronic":[21],"carrying":[23],"two":[24,96,122,420,438],"transgenes":[25,124,446],"linked":[26],"by":[27,136],"an":[28],"internal":[29],"ribosomal":[30],"entry":[31],"site":[32],"(IRES)":[33],"are":[34],"the":[35,50,55,75,164,175,199,202,247,279,291,310,353,397,427,464,498],"most":[36],"commonly":[37],"employed":[38],"accomplish":[41],"this":[42,67,345,404,433],"goal.":[43],"We":[44],"and":[45,150,161,179,185,213,256,267,297,325,367,392,450,463,500],"others":[46],"have":[47,436],"found":[48],"that":[49,93,274,336,407,417,440,453,477],"protein":[51,128,147],"encoded":[52],"downstream":[53],"IRES":[56,76],"these":[58,283,337],"not":[61],"expressed.":[63],"The":[64,171,209,232,401,470],"purpose":[65],"study":[68,405],"was":[69,134,221,236,307,319,327,358,375,387],"determine":[71],"if":[72],"replacement":[73],"our":[78,113,194,242,350],"self-inactivating,":[79],"insulated,":[80,116],"lentiviral":[81,108,117,415,475],"with":[83,288,316,360,372,384],"a":[84,91,126,225,272,333,423],"second,":[85],"independent,":[86],"cell-specific":[87,424],"promoter":[88,107,154,252,294,347,357,414,429],"would":[89],"produce":[90],"vector":[92,120,452,476],"expressed":[95,153,355],"proteins":[97,421],"retinal":[100],"cells":[101,324,391,395,449,462,490],"vivo.":[103],"METHODS:":[104],"Five":[105],"dual":[106,251,293,346,413,428],"were":[110,159,177,215,299],"constructed":[111],"using":[112,189],"self-inactivating":[114],"(SIN),":[115],"backbone.":[118],"Each":[119],"carried":[121],"independent":[123],"encoding":[125],"fluorescent":[127,190],"(GFP":[129],"or":[130,363,488],"tdTomato)":[131],"whose":[132],"expression":[133,200,280,342,443,456,482],"driven":[135],"three":[137,203,250,259],"photoreceptor":[138,204],"promoters":[139,205,338],"(interphotoreceptor":[140],"retinoid":[141],"binding":[142],"protein-IRPB1783;":[143],"guanylate":[144],"cyclase":[145],"activating":[146],"1-GCAP292;":[148],"rhodopsin-mOP500)":[149],"one":[151,451,458,474],"ubiquitously":[152,354],"(elongation":[155],"factor":[156],"1alpha-EF1alpha).":[157],"Constructs":[158],"packaged":[160],"injected":[162],"into":[163],"optic":[165],"vesicles":[166],"chicken":[169,207],"embryos.":[170],"day":[172],"before":[173],"hatching,":[174],"retinas":[176,286,314,370,382],"removed":[178],"examined":[180],"as":[181,186],"whole":[182],"mount":[183],"tissues":[184],"frozen":[187],"sections":[188],"microscopy.":[191],"RESULTS:":[192],"In":[193,241,285,313,349,369,381],"first":[195],"experiment,":[196,244,352],"we":[197,245,435],"characterized":[198,246],"retina.":[208,312,399],"activities":[210],"GCAP292":[212,220,362],"IRBP1783":[214],"restricted":[216,237],"cone":[218,303,323,379,448,461],"cells.":[219,231,240,304,380,469],"also":[222],"active":[223],"small":[226],"sub-group":[227],"inner":[229,311,398],"nuclear":[230],"activity":[233,248],"mOP500":[235,364],"rod":[239,331,390,468],"second":[243],"vectors:":[253],"GCAP292-GFP-IRBP1783-tdTomato,":[254],"IRBP-tdTomato-GCAP292-GFP,":[255],"IRBP1783-tdTomato-mOP500-GFP.":[257],"All":[258],"produced":[261],"easily":[262],"detectable":[263],"levels":[264],"GFP":[266,296,306,326,374,386],"tdTomato":[268,298,318],"transduced":[270],"retinas,":[271],"result":[273,334],"prompted":[275],"further":[276],"analyses":[277],"characteristics":[281],"vectors.":[284],"treated":[287,315,371,383],"either":[289,361],"GCAP292/IRBP1783":[292],"vectors,":[295],"only":[300,321,329,376],"detected":[301,308,320,328,377,388],"No":[305],"IRBP1783-tdTomato-mOP500-GFP,":[317],"cells,":[332],"indicating":[335],"retained":[339],"their":[340],"intrinsic":[341],"specificities":[343],"vector.":[348],"final":[351],"EF1alpha":[356],"paired":[359],"creating":[365],"EF1alpha-tdTomato-GCAP292-GFP":[366],"EF1alpha-tdTomato-mOP-GFP.":[368],"EF1alpha-tdTomato-GCAP292-GFP,":[373],"EF1alpha-tdTomato-mOP500-GFP,":[385],"several":[394],"within":[396],"CONCLUSIONS:":[400],"results":[402],"show":[406],"it":[408],"possible":[410],"construct":[412],"express":[419],"manner.":[425],"Among":[426],"created":[431],"for":[432],"study,":[434],"identified":[437],"specifically":[441,454],"target":[442],"both":[445],"targets":[455],"transgene":[459,466],"other":[465],"ability":[471],"create":[473],"targeting":[481],"single":[487],"vivo":[492],"should":[493],"prove":[494],"very":[495],"useful":[496],"development":[499],"delivery":[501],"complex,":[503],"combination":[504],"therapies":[505],"diseased":[507],"tissues.":[508]},"counts_by_year":[{"year":2021,"cited_by_count":1},{"year":2015,"cited_by_count":1},{"year":2014,"cited_by_count":2},{"year":2013,"cited_by_count":3},{"year":2012,"cited_by_count":3}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2016-06-24T00:00:00"}