{"id":"https://openalex.org/W2000475446","doi":"https://doi.org/10.5301/ejo.2011.8140","title":"Oral synthetic cis-retinoid therapy in subjects with Leber congenital amaurosis (LCA) due to lecithin:retinol acyltransferase (LRAT) or retinal pigment epithelial 65 protein (RPE65) mutations: preliminary results of a phase Ib open label trial","display_name":"Oral synthetic cis-retinoid therapy in subjects with Leber congenital amaurosis (LCA) due to lecithin:retinol acyltransferase (LRAT) or retinal pigment epithelial 65 protein (RPE65) mutations: preliminary results of a phase Ib open label trial","publication_year":2011,"publication_date":"2011-05-01","ids":{"openalex":"https://openalex.org/W2000475446","doi":"https://doi.org/10.5301/ejo.2011.8140","mag":"2000475446"},"language":"en","primary_location":{"id":"mag:2000475446","doi":"https://doi.org/10.5301/ejo.2011.8140","is_oa":false,"landing_page_url":"http://www.eur-j-ophthalmol.com/article/oral-synthetic-cis-retinoid-therapy-in-subjects-with-leber-congenital-amaurosis--lca--due-to-lecithin-retinol-acyltransferase--lrat--or-retinal-pigmen-ep-ret-660","pdf_url":null,"source":{"id":"https://openalex.org/S128637052","display_name":"European Journal of Ophthalmology","issn_l":"1120-6721","issn":["1120-6721","1724-6016"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310320017","host_organization_name":"SAGE Publishing","host_organization_lineage":["https://openalex.org/P4310320017"],"host_organization_lineage_names":["SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":null,"is_accepted":false,"is_published":false,"raw_source_name":"European Journal of Ophthalmology","raw_type":null},"volume":null,"type":"article","indexed_in":[],"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/A5084260018","display_name":"Robert Karel Koenekoop","orcid":"https://orcid.org/0000-0001-6697-0411","observed_orcids":["https://orcid.org/0000-0001-6697-0411"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Robert K. Koenekoop","raw_affiliation_strings":[],"raw_orcid":"https://orcid.org/0000-0001-6697-0411","affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5028853189","display_name":"Julie Racine","orcid":"https://orcid.org/0000-0003-4409-0936","observed_orcids":["https://orcid.org/0000-0003-4409-0936"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Julie Racine","raw_affiliation_strings":[],"raw_orcid":"https://orcid.org/0000-0003-4409-0936","affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5023070596","display_name":"S. Al Humaid","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"S. Al Humaid","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5042566435","display_name":"Ruifang Sui","orcid":"https://orcid.org/0000-0002-6659-0926","observed_orcids":["https://orcid.org/0000-0002-6659-0926","https://orcid.org/0000-0003-0115-7838"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Ruifang Sui","raw_affiliation_strings":[],"raw_orcid":"https://orcid.org/0000-0002-6659-0926","affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5013704008","display_name":"Elias I. Traboulsi","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"EI Traboulsi","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5040130510","display_name":"Juliana Maria Ferraz Sallum","orcid":"https://orcid.org/0000-0002-7206-4447","observed_orcids":["https://orcid.org/0000-0002-7206-4447"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Juliana Maria Ferraz Sallum","raw_affiliation_strings":[],"raw_orcid":"https://orcid.org/0000-0002-7206-4447","affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111044632","display_name":"D Panigrahi","orcid":"https://orcid.org/0009-0008-3972-1864","observed_orcids":["https://orcid.org/0009-0008-3972-1864"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"D Panigrahi","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111761652","display_name":"Krzysztof Palczewski","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"K. Palczewski","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"last","author":{"id":"https://openalex.org/A5073856478","display_name":"David A. Saperstein","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"David A. Saperstein","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]}],"institutions":[],"countries_distinct_count":0,"institutions_distinct_count":0,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":{"value":4150,"currency":"USD","value_usd":4150},"apc_paid":null,"fwci":0.1176,"has_fulltext":false,"cited_by_count":1,"citation_normalized_percentile":{"value":0.47754272,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":89,"max":94},"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.9940999746322632,"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":0.9940999746322632,"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/T10170","display_name":"Retinal Diseases and Treatments","score":0.0012000000569969416,"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/T11559","display_name":"Retinoids in leukemia and cellular processes","score":0.0008999999845400453,"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/leber-congenital-amaurosis","display_name":"Leber congenital amaurosis","score":0.9679999947547913},{"id":"https://openalex.org/keywords/lecithin-retinol-acyltransferase","display_name":"LRAT","score":0.8650000095367432},{"id":"https://openalex.org/keywords/rpe65-mutations","display_name":"RPE65 mutations","score":0.8349999785423279},{"id":"https://openalex.org/keywords/retinoid-therapy","display_name":"retinoid therapy","score":0.6769999861717224},{"id":"https://openalex.org/keywords/retinal-degeneration","display_name":"retinal degeneration","score":0.7059999704360962}],"concepts":[{"id":"https://openalex.org/C2780724529","wikidata":"https://www.wikidata.org/wiki/Q18031223","display_name":"RPE65","level":4,"score":0.9794172048568726},{"id":"https://openalex.org/C2775954498","wikidata":"https://www.wikidata.org/wiki/Q420418","display_name":"Retinoid","level":4,"score":0.7823562026023865},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.6024940013885498},{"id":"https://openalex.org/C2780827179","wikidata":"https://www.wikidata.org/wiki/Q422001","display_name":"Retinal","level":2,"score":0.5404875874519348},{"id":"https://openalex.org/C2775970874","wikidata":"https://www.wikidata.org/wiki/Q2609152","display_name":"Acyltransferase","level":3,"score":0.4872523844242096},{"id":"https://openalex.org/C2910190745","wikidata":"https://www.wikidata.org/wiki/Q5122112","display_name":"Cis-trans-Isomerases","level":5,"score":0.46020668745040894},{"id":"https://openalex.org/C118487528","wikidata":"https://www.wikidata.org/wiki/Q161437","display_name":"Ophthalmology","level":1,"score":0.4403700828552246},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.401703417301178},{"id":"https://openalex.org/C2781121885","wikidata":"https://www.wikidata.org/wiki/Q425517","display_name":"Retinoic acid","level":3,"score":0.36000463366508484},{"id":"https://openalex.org/C134018914","wikidata":"https://www.wikidata.org/wiki/Q162606","display_name":"Endocrinology","level":1,"score":0.34238988161087036},{"id":"https://openalex.org/C2778024200","wikidata":"https://www.wikidata.org/wiki/Q2346242","display_name":"Retinal pigment epithelium","level":3,"score":0.2832633852958679},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.2573784589767456},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.2049904763698578},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.19166404008865356},{"id":"https://openalex.org/C2910677434","wikidata":"https://www.wikidata.org/wiki/Q426411","display_name":"Peptidylprolyl isomerase","level":4,"score":0.0},{"id":"https://openalex.org/C2781264208","wikidata":"https://www.wikidata.org/wiki/Q118026","display_name":"Isomerase","level":3,"score":0.0}],"mesh":[],"locations_count":1,"locations":[{"id":"mag:2000475446","doi":"https://doi.org/10.5301/ejo.2011.8140","is_oa":false,"landing_page_url":"http://www.eur-j-ophthalmol.com/article/oral-synthetic-cis-retinoid-therapy-in-subjects-with-leber-congenital-amaurosis--lca--due-to-lecithin-retinol-acyltransferase--lrat--or-retinal-pigmen-ep-ret-660","pdf_url":null,"source":{"id":"https://openalex.org/S128637052","display_name":"European Journal of Ophthalmology","issn_l":"1120-6721","issn":["1120-6721","1724-6016"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-70","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310320017","host_organization_name":"SAGE Publishing","host_organization_lineage":["https://openalex.org/P4310320017"],"host_organization_lineage_names":["SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":null,"is_accepted":false,"is_published":null,"raw_source_name":"European Journal of Ophthalmology","raw_type":null}],"best_oa_location":null,"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":0.9476000070571899}],"sustainable_development_goals_aurora":[{"id":"https://metadata.un.org/sdg/2","display_name":"Zero hunger","score":0.5600000023841858}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W2070505320","https://openalex.org/W1971875600","https://openalex.org/W2126515199","https://openalex.org/W993875806","https://openalex.org/W2112741729","https://openalex.org/W2292872895","https://openalex.org/W2036702743","https://openalex.org/W2051399511","https://openalex.org/W2129617936","https://openalex.org/W2591991567","https://openalex.org/W2011930381","https://openalex.org/W2147711234","https://openalex.org/W2567791144","https://openalex.org/W1964728628","https://openalex.org/W3011618759","https://openalex.org/W2233403406","https://openalex.org/W28097838","https://openalex.org/W2067259137","https://openalex.org/W2552520284","https://openalex.org/W2066505838"],"abstract_inverted_index":null,"counts_by_year":[{"year":2012,"cited_by_count":1}],"updated_date":"2026-10-14T16:39:01.820549","created_date":"2025-10-10T00:00:00"}
