{"id":"https://openalex.org/W2013588839","doi":"https://doi.org/10.1167/iovs.10-6138","title":"Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis","display_name":"Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis","publication_year":2010,"publication_date":"2010-12-17","ids":{"openalex":"https://openalex.org/W2013588839","doi":"https://doi.org/10.1167/iovs.10-6138","mag":"2013588839","pmid":"https://pubmed.ncbi.nlm.nih.gov/21169527","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/3053305"},"language":"en","primary_location":{"id":"doi:10.1167/iovs.10-6138","doi":"https://doi.org/10.1167/iovs.10-6138","is_oa":false,"landing_page_url":"https://doi.org/10.1167/iovs.10-6138","pdf_url":null,"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":"green","oa_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/3053305","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5100445596","display_name":"Xia Li","orcid":"https://orcid.org/0000-0002-9794-2648","observed_orcids":["https://orcid.org/0000-0002-9794-2648","https://orcid.org/0000-0002-4691-4348","https://orcid.org/0000-0002-2991-6544"]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Xia Li","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100653587","display_name":"Wensheng Li","orcid":"https://orcid.org/0000-0002-1172-3349","observed_orcids":["https://orcid.org/0000-0002-1172-3349"]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Wensheng Li","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108849326","display_name":"Xufeng Dai","orcid":"https://orcid.org/0000-0001-8244-2248","observed_orcids":["https://orcid.org/0000-0001-8244-2248"]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Xufeng Dai","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5101775987","display_name":"Fansheng Kong","orcid":"https://orcid.org/0000-0002-0657-6823","observed_orcids":["https://orcid.org/0000-0002-0657-6823"]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Fansheng Kong","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5103909079","display_name":"Qinxiang Zheng","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Qinxiang Zheng","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5016372231","display_name":"Xiangtian Zhou","orcid":"https://orcid.org/0000-0002-1115-561X","observed_orcids":["https://orcid.org/0000-0002-1115-561X","https://orcid.org/0000-0003-0350-1356"]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Xiangtian Zhou","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100371042","display_name":"Fan L\u00fc","orcid":"https://orcid.org/0000-0002-8794-6944","observed_orcids":["https://orcid.org/0000-0002-8794-6944","https://orcid.org/0009-0006-0220-8982"]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Fan L\u00fc","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5053055285","display_name":"Bo Chang","orcid":"https://orcid.org/0000-0001-8259-7290","observed_orcids":["https://orcid.org/0000-0001-8259-7290"]},"institutions":[{"id":"https://openalex.org/I4210115256","display_name":"Jackson Laboratory","ror":"https://ror.org/021sy4w91","country_code":"US","type":"nonprofit","lineage":["https://openalex.org/I4210115256"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Bo Chang","raw_affiliation_strings":["The Jackson Laboratory, Bar Harbor, Maine;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"The Jackson Laboratory, Bar Harbor, Maine;","institution_ids":["https://openalex.org/I4210115256"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5055321629","display_name":"B\u00e4rbel Rohrer","orcid":"https://orcid.org/0000-0003-2758-3771","observed_orcids":["https://orcid.org/0000-0003-2758-3771"]},"institutions":[{"id":"https://openalex.org/I153297377","display_name":"Medical University of South Carolina","ror":"https://ror.org/012jban78","country_code":"US","type":"education","lineage":["https://openalex.org/I153297377"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"B\u00e4rbel Rohrer","raw_affiliation_strings":["the Department of Ophthalmology and 4Neurosciences Division of Research, Medical University of South Carolina, Charleston, South Carolina; and","the Department of Ophthalmology and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"the Department of Ophthalmology and 4Neurosciences Division of Research, Medical University of South Carolina, Charleston, South Carolina; and","institution_ids":["https://openalex.org/I153297377"]},{"raw_affiliation_string":"the Department 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":[{"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":true,"raw_author_name":"William. W. Hauswirth","raw_affiliation_strings":["the Department of Ophthalmology, University of Florida, Gainesville, Florida"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"the Department of Ophthalmology, University of Florida, Gainesville, Florida","institution_ids":["https://openalex.org/I33213144"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5101869619","display_name":"Jia Qu","orcid":"https://orcid.org/0000-0001-6360-2568","observed_orcids":["https://orcid.org/0000-0001-6360-2568"]},"institutions":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Jia Qu","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China;","institution_ids":["https://openalex.org/I27781120"]}]},{"author_position":"last","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":[{"id":"https://openalex.org/I27781120","display_name":"Wenzhou Medical University","ror":"https://ror.org/00rd5t069","country_code":"CN","type":"education","lineage":["https://openalex.org/I27781120"]},{"id":"https://openalex.org/I2800717037","display_name":"University of Florida Health","ror":"https://ror.org/04tk2gy88","country_code":"US","type":"education","lineage":["https://openalex.org/I2800717037","https://openalex.org/I33213144"]},{"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":["CN","US"],"is_corresponding":true,"raw_author_name":"Ji-jing Pang","raw_affiliation_strings":["From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China; the 5Department of Ophthalmology, University of Florida, Gainesville, Florida","the Department of Ophthalmology, University of Florida, Gainesville, Florida"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"From the Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical College, Wenzhou, PR China; the 5Department of Ophthalmology, University of Florida, Gainesville, Florida","institution_ids":["https://openalex.org/I27781120","https://openalex.org/I2800717037","https://openalex.org/I33213144"]},{"raw_affiliation_string":"the Department of Ophthalmology, University of Florida, Gainesville, Florida","institution_ids":["https://openalex.org/I33213144"]}]}],"institutions":[],"countries_distinct_count":2,"institutions_distinct_count":5,"corresponding_author_ids":["https://openalex.org/A5001100231","https://openalex.org/A5016372231","https://openalex.org/A5053055285","https://openalex.org/A5055321629","https://openalex.org/A5086264524","https://openalex.org/A5100371042","https://openalex.org/A5100445596","https://openalex.org/A5100653587","https://openalex.org/A5101775987","https://openalex.org/A5101869619","https://openalex.org/A5103909079","https://openalex.org/A5108849326"],"corresponding_institution_ids":["https://openalex.org/I153297377","https://openalex.org/I27781120","https://openalex.org/I2800717037","https://openalex.org/I33213144","https://openalex.org/I4210115256"],"apc_list":{"value":1850,"currency":"USD","value_usd":1850},"apc_paid":null,"fwci":2.3432,"has_fulltext":false,"cited_by_count":65,"citation_normalized_percentile":{"value":0.88144909,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":93,"max":99},"biblio":{"volume":"52","issue":"1","first_page":"7","last_page":"7"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.9984999895095825,"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":0.9984999895095825,"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":0.0006000000284984708,"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/T10170","display_name":"Retinal Diseases and Treatments","score":0.00019999999494757503,"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"}}],"keywords":[{"id":"https://openalex.org/keywords/rpe65","display_name":"RPE65","score":0.8560000061988831},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7599999904632568},{"id":"https://openalex.org/keywords/cone-degeneration","display_name":"cone degeneration","score":0.9190000295639038},{"id":"https://openalex.org/keywords/m-cones","display_name":"M-cones","score":0.7649999856948853},{"id":"https://openalex.org/keywords/s-cones","display_name":"S-cones","score":0.8880000114440918},{"id":"https://openalex.org/keywords/cone-opsin","display_name":"cone opsin","score":0.6489999890327454},{"id":"https://openalex.org/keywords/retinal-pigment-epithelium","display_name":"retinal pigment epithelium","score":0.8569999933242798},{"id":"https://openalex.org/keywords/treatment-delay","display_name":"treatment delay","score":0.800000011920929},{"id":"https://openalex.org/keywords/electroretinography","display_name":"electroretinogram","score":0.597000002861023},{"id":"https://openalex.org/keywords/vision-loss","display_name":"vision loss","score":0.3720000088214874}],"concepts":[{"id":"https://openalex.org/C2780724529","wikidata":"https://www.wikidata.org/wiki/Q18031223","display_name":"RPE65","level":4,"score":0.9353353977203369},{"id":"https://openalex.org/C30028475","wikidata":"https://www.wikidata.org/wiki/Q24785022","display_name":"Opsin","level":4,"score":0.8238471746444702},{"id":"https://openalex.org/C131667965","wikidata":"https://www.wikidata.org/wiki/Q445081","display_name":"Visual phototransduction","level":3,"score":0.6274102926254272},{"id":"https://openalex.org/C2781040256","wikidata":"https://www.wikidata.org/wiki/Q7316761","display_name":"Retinal degeneration","level":3,"score":0.6082366704940796},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5169495940208435},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.42023569345474243},{"id":"https://openalex.org/C2777093970","wikidata":"https://www.wikidata.org/wiki/Q169342","display_name":"Retina","level":2,"score":0.4153841733932495},{"id":"https://openalex.org/C2780827179","wikidata":"https://www.wikidata.org/wiki/Q422001","display_name":"Retinal","level":2,"score":0.35594192147254944},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.3367128372192383},{"id":"https://openalex.org/C2778024200","wikidata":"https://www.wikidata.org/wiki/Q2346242","display_name":"Retinal pigment epithelium","level":3,"score":0.3002512454986572},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.2515507638454437},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.1869795322418213},{"id":"https://openalex.org/C169760540","wikidata":"https://www.wikidata.org/wiki/Q207011","display_name":"Neuroscience","level":1,"score":0.18683013319969177},{"id":"https://openalex.org/C202033177","wikidata":"https://www.wikidata.org/wiki/Q423107","display_name":"Rhodopsin","level":3,"score":0.07855969667434692},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.07773619890213013}],"mesh":[{"descriptor_ui":"D000099178","descriptor_name":"Retinoid Isomerohydrolase","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"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":"D002352","descriptor_name":"Carrier Proteins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D004195","descriptor_name":"Disease Models, Animal","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D004596","descriptor_name":"Electroretinography","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":true},{"descriptor_ui":"D005822","descriptor_name":"Genetic Vectors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008810","descriptor_name":"Mice, Inbred C57BL","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008817","descriptor_name":"Mice, Mutant Strains","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008856","descriptor_name":"Microscopy, Fluorescence","qualifier_ui":null,"qualifier_name":null,"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":"D019084","descriptor_name":"Fluorescent Antibody Technique, Indirect","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D019745","descriptor_name":"cis-trans-Isomerases","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":"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":"D055358","descriptor_name":"Cone Opsins","qualifier_ui":"Q000378","qualifier_name":"metabolism","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":"Q000378","qualifier_name":"metabolism","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":false}],"locations_count":4,"locations":[{"id":"doi:10.1167/iovs.10-6138","doi":"https://doi.org/10.1167/iovs.10-6138","is_oa":false,"landing_page_url":"https://doi.org/10.1167/iovs.10-6138","pdf_url":null,"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:3053305","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/3053305","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:21169527","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/21169527","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"},{"id":"pmh:oai:mouseion.jax.org:stfb2011-1144","doi":null,"is_oa":false,"landing_page_url":"http://www.iovs.org/content/52/1/7.abstract","pdf_url":null,"source":{"id":"https://openalex.org/S4377196421","display_name":"The Mouseion at the JAXlibrary (Jackson Laboratory)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I4210115256","host_organization_name":"Jackson Laboratory","host_organization_lineage":["https://openalex.org/I4210115256"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Faculty Research 2011","raw_type":"text"}],"best_oa_location":{"id":"pmh:oai:pubmedcentral.nih.gov:3053305","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/3053305","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"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.9537000060081482}],"sustainable_development_goals_aurora":[{"display_name":"Life on land","id":"https://metadata.un.org/sdg/15","score":0.5799999833106995}],"study_designs":[],"awards":[{"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/G1808696035","display_name":"Alternative Pathway of Complement Activation in Age-Related Macular Degeneration","funder_award_id":"R01EY019320","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G2324864901","display_name":"Core Resources for Vision Research at MUSC","funder_award_id":"R24EY014793","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G3213560301","display_name":null,"funder_award_id":"N01 AA021004","funder_id":"https://openalex.org/F4320337611","funder_display_name":"Eunice Kennedy Shriver National Institute of Child Health and Human Development"},{"id":"https://openalex.org/G3666625135","display_name":null,"funder_award_id":"EY 14793","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G4815552212","display_name":null,"funder_award_id":"EY018331","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G552440617","display_name":null,"funder_award_id":"R01-EY019943","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"},{"id":"https://openalex.org/G7166388802","display_name":"Eye Mutant Resource","funder_award_id":"5r01ey019943-13","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G7820020350","display_name":"Nanoparticle & AAV Approaches to Rapid Onset, Stable Retinal Gene Therapy","funder_award_id":"R21EY018331","funder_id":"https://openalex.org/F4320337350","funder_display_name":"National Eye Institute"}],"funders":[{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320337350","display_name":"National Eye Institute","ror":"https://ror.org/03wkg3b53"},{"id":"https://openalex.org/F4320337611","display_name":"Eunice Kennedy Shriver National Institute of Child Health and Human Development","ror":"https://ror.org/04byxyr05"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":49,"referenced_works":["https://openalex.org/W1488105258","https://openalex.org/W1490065438","https://openalex.org/W1531497333","https://openalex.org/W1578849205","https://openalex.org/W1587435244","https://openalex.org/W1623115833","https://openalex.org/W1968667617","https://openalex.org/W1968853259","https://openalex.org/W1972258703","https://openalex.org/W1976599692","https://openalex.org/W1978131598","https://openalex.org/W1981432109","https://openalex.org/W1983421509","https://openalex.org/W1985844587","https://openalex.org/W1992118354","https://openalex.org/W1992618284","https://openalex.org/W1995140705","https://openalex.org/W1995284319","https://openalex.org/W1999109674","https://openalex.org/W2002626096","https://openalex.org/W2016170285","https://openalex.org/W2024740152","https://openalex.org/W2030588688","https://openalex.org/W2034261396","https://openalex.org/W2034379083","https://openalex.org/W2039358746","https://openalex.org/W2048809756","https://openalex.org/W2056320636","https://openalex.org/W2062618306","https://openalex.org/W2070893919","https://openalex.org/W2081064825","https://openalex.org/W2082177594","https://openalex.org/W2085854897","https://openalex.org/W2086546231","https://openalex.org/W2094430279","https://openalex.org/W2100522480","https://openalex.org/W2100821349","https://openalex.org/W2102131183","https://openalex.org/W2105726171","https://openalex.org/W2129898494","https://openalex.org/W2135558903","https://openalex.org/W2137222303","https://openalex.org/W2141354154","https://openalex.org/W2146847499","https://openalex.org/W2151809142","https://openalex.org/W2154702270","https://openalex.org/W2188903576","https://openalex.org/W2323692398","https://openalex.org/W2432787123"],"related_works":["https://openalex.org/W3204104734","https://openalex.org/W1976599692","https://openalex.org/W1968412229","https://openalex.org/W4238746605","https://openalex.org/W1180586538","https://openalex.org/W2010207266","https://openalex.org/W1995284319","https://openalex.org/W1968667617","https://openalex.org/W2019718689","https://openalex.org/W2010690970"],"abstract_inverted_index":{"PURPOSE:":[0],"RPE65":[1,35,46,80],"function":[2,172,242],"is":[3],"necessary":[4],"in":[5,20,98,166,206],"the":[6,55,95,109,156,178,194,219,241,246,255],"retinal":[7],"pigment":[8],"epithelium":[9],"(RPE)":[10],"to":[11,92],"generate":[12],"chromophore":[13],"for":[14,155,254],"all":[15],"opsins.":[16],"Its":[17],"absence":[18],"results":[19,213,250],"vision":[21,44],"loss":[22],"and":[23,118,126,136,145,170,173,181,215,243],"rapid":[24],"cone":[25,65,85,127,137,158,209,228],"degeneration.":[26,86],"Recent":[27],"Leber":[28],"congenital":[29],"amaurosis":[30],"type":[31],"2":[32,121],"(LCA":[33],"with":[34,108,148,208],"mutations)":[36],"phase":[37],"I":[38],"clinical":[39,258],"trials":[40],"demonstrated":[41],"restoration":[42],"of":[43,62,196,218,245],"on":[45],"gene":[47,197,223],"transfer":[48],"into":[49],"RPE":[50],"cells":[51,149,187,207],"overlying":[52],"cones.":[53,248],"In":[54],"rd12":[56,100],"mouse,":[57],"a":[58],"naturally":[59],"occurring":[60],"model":[61],"RPE65-LCA":[63],"early":[64,84,227],"degeneration":[66,142],"was":[67,90,112,129],"observed;":[68],"however,":[69],"some":[70],"peripheral":[71],"M-cones":[72],"remained.":[73],"A":[74],"prior":[75],"study":[76,89,221],"showed":[77],"that":[78,154,200,222,236],"AAV-mediated":[79],"expression":[81,202],"can":[82,102,225,239],"prevent":[83],"The":[87,212],"present":[88,192],"conducted":[91],"test":[93],"whether":[94],"remaining":[96,179,247],"cones":[97],"older":[99],"mice":[101],"be":[103,204],"rescued.":[104],"METHODS:":[105],"Subretinal":[106],"treatment":[107,238],"scAAV5-smCBA-hRPE65":[110],"vector":[111],"initiated":[113],"at":[114,163,193],"postnatal":[115],"day":[116],"(P)14":[117],"P90.":[119],"After":[120],"months,":[122],"electroretinograms":[123],"were":[124,188,191],"recorded,":[125],"morphology":[128,244],"analyzed":[130],"by":[131],"using":[132],"cone-specific":[133],"peanut":[134],"agglutinin":[135],"opsin-specific":[138],"antibodies.":[139],"RESULTS:":[140],"Cone":[141],"started":[143],"centrally":[144],"spread":[146],"ventrally,":[147],"losing":[150],"cone-opsin":[151],"staining":[152],"before":[153],"PNA-lectin-positive":[157],"sheath.":[159],"Gene":[160],"therapy":[161,224],"starting":[162],"P14":[164],"resulted":[165],"almost":[167],"wild-type":[168],"M-":[169],"S-cone":[171],"morphology.":[174],"Delaying":[175],"gene-replacement":[176],"rescued":[177],"M-cones,":[180],"most":[182],"important,":[183,232],"more":[184,231],"M-cone":[185],"opsin-positive":[186],"identified":[189],"than":[190],"onset":[195],"therapy,":[198],"suggesting":[199],"opsin":[201],"could":[203],"reinitiated":[205],"sheaths.":[210],"CONCLUSIONS:":[211],"support":[214],"extend":[216],"those":[217],"previous":[220],"stop":[226],"degeneration,":[229],"and,":[230],"they":[233],"provide":[234],"proof":[235],"delayed":[237],"restore":[240],"These":[249],"have":[251],"important":[252],"implications":[253],"ongoing":[256],"LCA2":[257],"trials.":[259]},"counts_by_year":[{"year":2026,"cited_by_count":2},{"year":2025,"cited_by_count":4},{"year":2023,"cited_by_count":2},{"year":2022,"cited_by_count":2},{"year":2021,"cited_by_count":3},{"year":2020,"cited_by_count":3},{"year":2019,"cited_by_count":4},{"year":2018,"cited_by_count":3},{"year":2017,"cited_by_count":7},{"year":2016,"cited_by_count":5},{"year":2015,"cited_by_count":7},{"year":2014,"cited_by_count":5},{"year":2013,"cited_by_count":6},{"year":2012,"cited_by_count":10}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2016-06-24T00:00:00"}
