{"id":"https://openalex.org/W1990641747","doi":"https://doi.org/10.1167/iovs.11-7867","title":"Long-term Preservation of Cone Photoreceptors and Restoration of Cone Function by Gene Therapy in the Guanylate Cyclase-1 Knockout (GC1KO) Mouse","display_name":"Long-term Preservation of Cone Photoreceptors and Restoration of Cone Function by Gene Therapy in the Guanylate Cyclase-1 Knockout (GC1KO) Mouse","publication_year":2011,"publication_date":"2011-07-21","ids":{"openalex":"https://openalex.org/W1990641747","doi":"https://doi.org/10.1167/iovs.11-7867","mag":"1990641747","pmid":"https://pubmed.ncbi.nlm.nih.gov/21778276","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/3207713"},"language":"en","primary_location":{"id":"doi:10.1167/iovs.11-7867","doi":"https://doi.org/10.1167/iovs.11-7867","is_oa":true,"landing_page_url":"https://doi.org/10.1167/iovs.11-7867","pdf_url":"https://iovs.arvojournals.org/arvo/content_public/journal/iovs/933248/z7g01011007098.pdf","source":{"id":"https://openalex.org/S76304953","display_name":"Investigative Ophthalmology & Visual Science","issn_l":"0146-0404","issn":["0146-0404","1552-5783"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310321950","host_organization_name":"Cadmus Press","host_organization_lineage":["https://openalex.org/P4310321950"],"host_organization_lineage_names":["Cadmus Press"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Investigative Opthalmology &amp; Visual Science","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"bronze","oa_url":"https://iovs.arvojournals.org/arvo/content_public/journal/iovs/933248/z7g01011007098.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5008889406","display_name":"Sanford L. Boye","orcid":"https://orcid.org/0000-0002-7312-3197","observed_orcids":["https://orcid.org/0000-0002-7312-3197"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Sanford L. Boye","raw_affiliation_strings":["From the Departments of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Departments of Ophthalmology and","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5014115936","display_name":"Thomas J. Conlon","orcid":"https://orcid.org/0009-0003-0658-8845","observed_orcids":["https://orcid.org/0009-0003-0658-8845"]},"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"]},{"id":"https://openalex.org/I4210147155","display_name":"Pediatrics and Genetics","ror":"https://ror.org/03t432d20","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210147155"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Thomas Conlon","raw_affiliation_strings":["Pediatrics and the 2Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida","Pediatrics and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Pediatrics and the 2Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida","institution_ids":["https://openalex.org/I33213144"]},{"raw_affiliation_string":"Pediatrics and","institution_ids":["https://openalex.org/I4210147155"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5052819930","display_name":"Kirsten E. Erger","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"]},{"id":"https://openalex.org/I4210147155","display_name":"Pediatrics and Genetics","ror":"https://ror.org/03t432d20","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210147155"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Kirsten Erger","raw_affiliation_strings":["Pediatrics and the 2Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida","Pediatrics and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Pediatrics and the 2Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida","institution_ids":["https://openalex.org/I33213144"]},{"raw_affiliation_string":"Pediatrics and","institution_ids":["https://openalex.org/I4210147155"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5051471378","display_name":"Renee C. Ryals","orcid":"https://orcid.org/0000-0002-7182-7238","observed_orcids":["https://orcid.org/0000-0002-7182-7238"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Renee Ryals","raw_affiliation_strings":["From the Departments of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Departments of Ophthalmology and","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5031564687","display_name":"Andy W. Neeley","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Andy Neeley","raw_affiliation_strings":["From the Departments of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Departments of Ophthalmology and","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5022886286","display_name":"Travis L. Cossette","orcid":"https://orcid.org/0000-0003-0398-5047","observed_orcids":["https://orcid.org/0000-0003-0398-5047"]},"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"]},{"id":"https://openalex.org/I4210147155","display_name":"Pediatrics and Genetics","ror":"https://ror.org/03t432d20","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210147155"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Travis Cossette","raw_affiliation_strings":["Pediatrics and the 2Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida","Pediatrics and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Pediatrics and the 2Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida","institution_ids":["https://openalex.org/I33213144"]},{"raw_affiliation_string":"Pediatrics and","institution_ids":["https://openalex.org/I4210147155"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5001100231","display_name":"Jijing Pang","orcid":"https://orcid.org/0000-0002-9235-7909","observed_orcids":["https://orcid.org/0000-0002-9235-7909"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Jijing Pang","raw_affiliation_strings":["From the Departments of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Departments of Ophthalmology and","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5028149469","display_name":"Frank M. Dyka","orcid":"https://orcid.org/0000-0001-7973-7125","observed_orcids":["https://orcid.org/0000-0001-7973-7125"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Frank M. Dyka","raw_affiliation_strings":["From the Departments of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Departments of Ophthalmology and","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5086264524","display_name":"William W. Hauswirth","orcid":"https://orcid.org/0000-0002-3244-4947","observed_orcids":["https://orcid.org/0000-0002-3244-4947"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"William W. Hauswirth","raw_affiliation_strings":["From the Departments of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Departments of Ophthalmology and","institution_ids":[]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5008889406","display_name":"Sanford L. Boye","orcid":"https://orcid.org/0000-0002-7312-3197","observed_orcids":["https://orcid.org/0000-0002-7312-3197"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Shannon E. Boye","raw_affiliation_strings":["From the Departments of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Departments of Ophthalmology and","institution_ids":[]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5001100231","https://openalex.org/A5008889406","https://openalex.org/A5014115936","https://openalex.org/A5022886286","https://openalex.org/A5028149469","https://openalex.org/A5031564687","https://openalex.org/A5051471378","https://openalex.org/A5052819930","https://openalex.org/A5086264524"],"corresponding_institution_ids":["https://openalex.org/I33213144","https://openalex.org/I4210147155"],"apc_list":{"value":1850,"currency":"USD","value_usd":1850},"apc_paid":null,"fwci":4.281,"has_fulltext":true,"cited_by_count":63,"citation_normalized_percentile":{"value":0.95509101,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":88,"max":99},"biblio":{"volume":"52","issue":"10","first_page":"7098","last_page":"7098"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.9771000146865845,"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"}},"topics":[{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.9771000146865845,"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/T10613","display_name":"Virus-based gene therapy research","score":0.019500000402331352,"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":0.0006000000284984708,"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/gene-therapy","display_name":"gene therapy","score":0.6700000166893005},{"id":"https://openalex.org/keywords/aav5","display_name":"AAV5","score":0.8920000195503235},{"id":"https://openalex.org/keywords/subretinal-delivery","display_name":"subretinal delivery","score":0.8460000157356262},{"id":"https://openalex.org/keywords/cone-photoreceptors","display_name":"cone photoreceptors","score":0.8539999723434448},{"id":"https://openalex.org/keywords/photoreceptor-survival","display_name":"photoreceptor survival","score":0.7570000290870667},{"id":"https://openalex.org/keywords/retinal-function","display_name":"retinal function","score":0.5360000133514404},{"id":"https://openalex.org/keywords/long-term-treatment","display_name":"long-term treatment","score":0.5709999799728394}],"concepts":[{"id":"https://openalex.org/C2777093970","wikidata":"https://www.wikidata.org/wiki/Q169342","display_name":"Retina","level":2,"score":0.6220410466194153},{"id":"https://openalex.org/C2779113765","wikidata":"https://www.wikidata.org/wiki/Q681562","display_name":"Electroretinography","level":3,"score":0.5789892077445984},{"id":"https://openalex.org/C182704531","wikidata":"https://www.wikidata.org/wiki/Q1364740","display_name":"Knockout mouse","level":3,"score":0.5397961735725403},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5322980880737305},{"id":"https://openalex.org/C2777102633","wikidata":"https://www.wikidata.org/wiki/Q735343","display_name":"Muller glia","level":4,"score":0.4726913273334503},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.4288780093193054},{"id":"https://openalex.org/C2780837183","wikidata":"https://www.wikidata.org/wiki/Q186029","display_name":"Optic nerve","level":2,"score":0.4196755886077881},{"id":"https://openalex.org/C169760540","wikidata":"https://www.wikidata.org/wiki/Q207011","display_name":"Neuroscience","level":1,"score":0.35503315925598145},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.3238784074783325},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.2472822070121765},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.15730682015419006},{"id":"https://openalex.org/C201750760","wikidata":"https://www.wikidata.org/wiki/Q1896218","display_name":"Progenitor cell","level":3,"score":0.0},{"id":"https://openalex.org/C28328180","wikidata":"https://www.wikidata.org/wiki/Q48196","display_name":"Stem cell","level":2,"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":"D002470","descriptor_name":"Cell Survival","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004195","descriptor_name":"Disease Models, Animal","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004596","descriptor_name":"Electroretinography","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","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":"D006162","descriptor_name":"Guanylate Cyclase","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D011956","descriptor_name":"Receptors, Cell Surface","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D012333","descriptor_name":"RNA, Messenger","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D017949","descriptor_name":"Retinal Cone Photoreceptor Cells","qualifier_ui":"Q000502","qualifier_name":"physiology","is_major_topic":true},{"descriptor_ui":"D018345","descriptor_name":"Mice, Knockout","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D020133","descriptor_name":"Reverse Transcriptase Polymerase Chain Reaction","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D020962","descriptor_name":"Heterotrimeric GTP-Binding 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":"D051606","descriptor_name":"G-Protein-Coupled Receptor Kinase 1","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D056965","descriptor_name":"Injections, Intraocular","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D057130","descriptor_name":"Leber Congenital Amaurosis","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D057130","descriptor_name":"Leber Congenital Amaurosis","qualifier_ui":"Q000503","qualifier_name":"physiopathology","is_major_topic":false},{"descriptor_ui":"D057130","descriptor_name":"Leber Congenital Amaurosis","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":true}],"locations_count":3,"locations":[{"id":"doi:10.1167/iovs.11-7867","doi":"https://doi.org/10.1167/iovs.11-7867","is_oa":true,"landing_page_url":"https://doi.org/10.1167/iovs.11-7867","pdf_url":"https://iovs.arvojournals.org/arvo/content_public/journal/iovs/933248/z7g01011007098.pdf","source":{"id":"https://openalex.org/S76304953","display_name":"Investigative Ophthalmology & Visual Science","issn_l":"0146-0404","issn":["0146-0404","1552-5783"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310321950","host_organization_name":"Cadmus Press","host_organization_lineage":["https://openalex.org/P4310321950"],"host_organization_lineage_names":["Cadmus Press"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Investigative Opthalmology &amp; Visual Science","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:3207713","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/3207713","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"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":"Invest Ophthalmol Vis Sci","raw_type":"Text"},{"id":"pmid:21778276","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/21778276","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":"Investigative ophthalmology & visual science","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1167/iovs.11-7867","doi":"https://doi.org/10.1167/iovs.11-7867","is_oa":true,"landing_page_url":"https://doi.org/10.1167/iovs.11-7867","pdf_url":"https://iovs.arvojournals.org/arvo/content_public/journal/iovs/933248/z7g01011007098.pdf","source":{"id":"https://openalex.org/S76304953","display_name":"Investigative Ophthalmology & Visual Science","issn_l":"0146-0404","issn":["0146-0404","1552-5783"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310321950","host_organization_name":"Cadmus Press","host_organization_lineage":["https://openalex.org/P4310321950"],"host_organization_lineage_names":["Cadmus Press"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Investigative Opthalmology &amp; Visual Science","raw_type":"journal-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":0.8560000061988831}],"sustainable_development_goals_aurora":[{"id":"https://metadata.un.org/sdg/15","display_name":"Life on land","score":0.6499999761581421}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1331656083","display_name":null,"funder_award_id":"EY 13729","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G162802300","display_name":"Vision Research Core","funder_award_id":"P30EY021721","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G1981988940","display_name":null,"funder_award_id":"EY 08571","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G2614672191","display_name":"Gene Therapy for Leber Congenital Amaurosis","funder_award_id":"U10EY013729","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"},{"id":"https://openalex.org/G686859034","display_name":"Retinal Gene Delivery by Adeno-Associated Virus","funder_award_id":"R01EY011123","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G823154836","display_name":null,"funder_award_id":"EY-11123","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"}],"funders":[{"id":"https://openalex.org/F4320306811","display_name":"Research to Prevent Blindness","ror":"https://ror.org/04drjs621"},{"id":"https://openalex.org/F4320308668","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60"},{"id":"https://openalex.org/F4320309460","display_name":"Macula Vision Research Foundation","ror":"https://ror.org/03cpssz74"},{"id":"https://openalex.org/F4320310009","display_name":"University of Florida","ror":"https://ror.org/02y3ad647"},{"id":"https://openalex.org/F4320310050","display_name":"University of Utah","ror":"https://ror.org/03r0ha626"},{"id":"https://openalex.org/F4320337350","display_name":"National Eye Institute","ror":"https://ror.org/03wkg3b53"}],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W1990641747.pdf","grobid_xml":"https://content.openalex.org/works/W1990641747.grobid-xml"},"referenced_works_count":54,"referenced_works":["https://openalex.org/W1550806901","https://openalex.org/W1555165926","https://openalex.org/W1914786390","https://openalex.org/W1967162995","https://openalex.org/W1968186746","https://openalex.org/W1968667617","https://openalex.org/W1984764475","https://openalex.org/W1985526748","https://openalex.org/W1989352995","https://openalex.org/W1989566939","https://openalex.org/W1992246699","https://openalex.org/W2004331116","https://openalex.org/W2008485465","https://openalex.org/W2010335307","https://openalex.org/W2022040013","https://openalex.org/W2024979469","https://openalex.org/W2026820540","https://openalex.org/W2029389016","https://openalex.org/W2037686336","https://openalex.org/W2037854344","https://openalex.org/W2040427946","https://openalex.org/W2040943511","https://openalex.org/W2042652151","https://openalex.org/W2043408047","https://openalex.org/W2044998344","https://openalex.org/W2045168845","https://openalex.org/W2048008983","https://openalex.org/W2048965038","https://openalex.org/W2049763116","https://openalex.org/W2053204895","https://openalex.org/W2057708342","https://openalex.org/W2061425681","https://openalex.org/W2064779400","https://openalex.org/W2084994296","https://openalex.org/W2087506885","https://openalex.org/W2093023074","https://openalex.org/W2093710097","https://openalex.org/W2107277218","https://openalex.org/W2111993777","https://openalex.org/W2113903634","https://openalex.org/W2122236774","https://openalex.org/W2136302254","https://openalex.org/W2143938161","https://openalex.org/W2148004446","https://openalex.org/W2151697358","https://openalex.org/W2165170253","https://openalex.org/W2171654274","https://openalex.org/W2172107348","https://openalex.org/W2314470532","https://openalex.org/W2992367859","https://openalex.org/W4240588598","https://openalex.org/W4241253251","https://openalex.org/W4243101475","https://openalex.org/W4249609410"],"related_works":["https://openalex.org/W2049763116","https://openalex.org/W2081866115","https://openalex.org/W2010763602","https://openalex.org/W1924768710","https://openalex.org/W2770652065","https://openalex.org/W1985526748","https://openalex.org/W1996875707","https://openalex.org/W2568562909","https://openalex.org/W2072440756","https://openalex.org/W947418538"],"abstract_inverted_index":{"PURPOSE:":[0],"The":[1,210],"authors":[2,50,211],"previously":[3],"showed":[4],"that":[5,59,217],"subretinal":[6],"delivery":[7],"of":[8,28,103,117,127,161,195,206,228,239,249],"AAV5":[9],"vectors":[10,57,140],"containing":[11],"murine":[12],"guanylate":[13],"cyclase-1":[14],"(GC1)":[15],"cDNA":[16],"driven":[17],"by":[18,54,100,125],"either":[19],"photoreceptor-specific":[20],"(hGRK1)":[21],"or":[22,82],"ubiquitous":[23],"(smCBA)":[24],"promoters":[25],"was":[26,84,98,123,136,163],"capable":[27],"restoring":[29],"cone-mediated":[30],"function":[31,97,135],"and":[32,35,69,94,107,114,130,185],"visual":[33],"behavior":[34],"preserving":[36],"cone":[37,108],"photoreceptors":[38,167],"in":[39,76,166,200,224],"the":[40,49,55,62,115,121,145,196,214,237,246],"GC1":[41,105,162,229],"knockout":[42],"(GC1KO)":[43],"mouse":[44],"for":[45,177,213,236,245],"3":[46],"months.":[47],"Here,":[48],"compared":[51],"therapy":[52,73,219,243],"conferred":[53],"aforementioned":[56],"to":[58,87,154,169],"achieved":[60],"with":[61,112,138,142],"highly":[63],"efficient":[64],"capsid":[65],"tyrosine":[66],"mutant":[67],"AAV8(Y733F)":[68,143],"asked":[70],"whether":[71],"long-term":[72,218],"is":[74,222],"achievable":[75,223],"this":[77],"model.":[78],"METHODS:":[79],"AAV5-hGRK1-mGC1,":[80],"AAV5-smCBA-mGC1,":[81],"AAV8(Y733F)-hGRK1-mGC1":[83],"delivered":[85],"subretinally":[86],"GC1KO":[88],"mice":[89],"between":[90],"postnatal":[91],"day":[92],"(P)14":[93],"P25.":[95],"Retinal":[96],"assayed":[99,111],"electroretinography.":[101],"Localization":[102],"AAV-mediated":[104,159],"expression":[106,160],"survival":[109],"were":[110,150,175,189],"immunohistochemistry,":[113],"spread":[116],"vector":[118,187,244],"genomes":[119,188],"beyond":[120],"retina":[122],"quantified":[124],"PCR":[126],"optic":[128,193],"nerve":[129,194],"brain":[131,204],"tissue.":[132],"RESULTS:":[133],"Cone":[134],"restored":[137],"all":[139],"tested,":[141],"being":[144],"most":[146],"efficient.":[147],"Electroretinographic":[148],"responses":[149],"clearly":[151],"measurable":[152],"out":[153,168],"1":[155],"year":[156],"after":[157,172,182],"treatment.":[158,183],"found":[164],"exclusively":[165],"15":[170],"months":[171,181],"injection.":[173],"Cones":[174],"preserved":[176],"at":[178],"least":[179],"11":[180],"AAV5-":[184],"AAV8(733)-delivered":[186],"recovered":[190],"primarily":[191],"from":[192,203],"treated":[197],"eye":[198],"and,":[199],"only":[201],"instance,":[202],"(1":[205],"20":[207],"samples).":[208],"CONCLUSIONS:":[209],"demonstrate":[212],"first":[215],"time":[216],"(\u223c1":[220],"year)":[221],"a":[225],"mammalian":[226],"model":[227],"deficiency.":[230],"These":[231],"data":[232],"provide":[233],"additional":[234],"justification":[235],"development":[238],"an":[240],"AAV-based":[241],"gene":[242],"clinical":[247],"treatment":[248],"Leber":[250],"congenital":[251],"amaurosis-1.":[252]},"counts_by_year":[{"year":2025,"cited_by_count":2},{"year":2023,"cited_by_count":2},{"year":2022,"cited_by_count":4},{"year":2021,"cited_by_count":1},{"year":2019,"cited_by_count":4},{"year":2018,"cited_by_count":5},{"year":2017,"cited_by_count":2},{"year":2016,"cited_by_count":5},{"year":2015,"cited_by_count":5},{"year":2014,"cited_by_count":10},{"year":2013,"cited_by_count":11},{"year":2012,"cited_by_count":10}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2016-06-24T00:00:00"}
