{"id":"https://openalex.org/W2743983049","doi":null,"title":"Evaluation of in silico reconstructed ancestral adeno-associated virus for gene augmentation therapy in a mouse model of LCA","display_name":"Evaluation of in silico reconstructed ancestral adeno-associated virus for gene augmentation therapy in a mouse model of LCA","publication_year":2017,"publication_date":"2017-06-23","ids":{"openalex":"https://openalex.org/W2743983049","mag":"2743983049"},"language":"en","primary_location":{"id":"mag:2743983049","doi":null,"is_oa":true,"landing_page_url":"https://iovs.arvojournals.org/article.aspx?articleid=2641228","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":true,"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":null,"is_accepted":false,"is_published":false,"raw_source_name":"Investigative Ophthalmology & Visual Science","raw_type":null},"volume":null,"type":"article","is_traditional":true,"indexed_in":[],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://iovs.arvojournals.org/article.aspx?articleid=2641228","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5082883573","display_name":"Sarah Wassmer","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1287163989","display_name":"Massachusetts Eye and Ear Infirmary","ror":"https://ror.org/04g3dn724","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1287163989"]},{"id":"https://openalex.org/I136199984","display_name":"Harvard University","ror":"https://ror.org/03vek6s52","country_code":"US","type":"education","lineage":["https://openalex.org/I136199984"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Sarah Wassmer","raw_affiliation_strings":["Ophthalmology, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Ophthalmology, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I1287163989","https://openalex.org/I136199984"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113464618","display_name":"Basil S. Pawlyk","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1287163989","display_name":"Massachusetts Eye and Ear Infirmary","ror":"https://ror.org/04g3dn724","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1287163989"]},{"id":"https://openalex.org/I136199984","display_name":"Harvard University","ror":"https://ror.org/03vek6s52","country_code":"US","type":"education","lineage":["https://openalex.org/I136199984"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Basil S. Pawlyk","raw_affiliation_strings":["Ophthalmology, Berman-Gund Laboratory, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Ophthalmology, Berman-Gund Laboratory, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I1287163989","https://openalex.org/I136199984"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5033478208","display_name":"Jessica Blake","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1287163989","display_name":"Massachusetts Eye and Ear Infirmary","ror":"https://ror.org/04g3dn724","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1287163989"]},{"id":"https://openalex.org/I136199984","display_name":"Harvard University","ror":"https://ror.org/03vek6s52","country_code":"US","type":"education","lineage":["https://openalex.org/I136199984"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jessica Blake","raw_affiliation_strings":["Ophthalmology, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Ophthalmology, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I1287163989","https://openalex.org/I136199984"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5091652585","display_name":"Michael A. Sandberg","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1287163989","display_name":"Massachusetts Eye and Ear Infirmary","ror":"https://ror.org/04g3dn724","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1287163989"]},{"id":"https://openalex.org/I136199984","display_name":"Harvard University","ror":"https://ror.org/03vek6s52","country_code":"US","type":"education","lineage":["https://openalex.org/I136199984"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Michael A. Sandberg","raw_affiliation_strings":["Ophthalmology, Berman-Gund Laboratory, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States","Ophthalmology, Harvard Medical School, Boston, Massachusetts, United States"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Ophthalmology, Berman-Gund Laboratory, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I1287163989","https://openalex.org/I136199984"]},{"raw_affiliation_string":"Ophthalmology, Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I136199984"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048366850","display_name":"Eric A. Pierce","orcid":"https://orcid.org/0000-0002-2354-4102","observed_orcids":["https://orcid.org/0000-0002-2354-4102"]},"institutions":[{"id":"https://openalex.org/I1287163989","display_name":"Massachusetts Eye and Ear Infirmary","ror":"https://ror.org/04g3dn724","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1287163989"]},{"id":"https://openalex.org/I136199984","display_name":"Harvard University","ror":"https://ror.org/03vek6s52","country_code":"US","type":"education","lineage":["https://openalex.org/I136199984"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Eric A. Pierce","raw_affiliation_strings":["Ophthalmology, Harvard Medical School, Boston, Massachusetts, United States","Ophthalmology, Ocular Genomics Institute, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States"],"raw_orcid":"https://orcid.org/0000-0002-2354-4102","affiliations":[{"raw_affiliation_string":"Ophthalmology, Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I136199984"]},{"raw_affiliation_string":"Ophthalmology, Ocular Genomics Institute, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I1287163989","https://openalex.org/I136199984"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5009839456","display_name":"Luk H. Vandenberghe","orcid":"https://orcid.org/0000-0002-3508-4924","observed_orcids":["https://orcid.org/0000-0002-3508-4924"]},"institutions":[{"id":"https://openalex.org/I1287163989","display_name":"Massachusetts Eye and Ear Infirmary","ror":"https://ror.org/04g3dn724","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1287163989"]},{"id":"https://openalex.org/I136199984","display_name":"Harvard University","ror":"https://ror.org/03vek6s52","country_code":"US","type":"education","lineage":["https://openalex.org/I136199984"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Luk H. Vandenberghe","raw_affiliation_strings":["Ophthalmology, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States"],"raw_orcid":"https://orcid.org/0000-0002-3508-4924","affiliations":[{"raw_affiliation_string":"Ophthalmology, Massachusetts Eye and Ear Infirmary / Harvard Medical School, Boston, Massachusetts, United States","institution_ids":["https://openalex.org/I1287163989","https://openalex.org/I136199984"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":{"value":1850,"currency":"USD","value_usd":1850},"apc_paid":{"value":1850,"currency":"USD","value_usd":1850},"fwci":0.0,"has_fulltext":false,"cited_by_count":1,"citation_normalized_percentile":{"value":0.0,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":88,"max":93},"biblio":{"volume":"58","issue":"8","first_page":"4086","last_page":"4086"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.5088000297546387,"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"}},"topics":[{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.5088000297546387,"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/T10613","display_name":"Virus-based gene therapy research","score":0.4706999957561493,"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":0.0024999999441206455,"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/adeno-associated-virus","display_name":"adeno-associated virus","score":0.9549999833106995},{"id":"https://openalex.org/keywords/gene-augmentation-therapy","display_name":"gene augmentation therapy","score":0.8119999766349792},{"id":"https://openalex.org/keywords/leber-congenital-amaurosis","display_name":"Leber congenital amaurosis","score":0.8849999904632568},{"id":"https://openalex.org/keywords/mouse-model","display_name":"mouse model","score":0.7879999876022339},{"id":"https://openalex.org/keywords/retinal-gene-therapy","display_name":"retinal gene therapy","score":0.7250000238418579}],"concepts":[{"id":"https://openalex.org/C2778107364","wikidata":"https://www.wikidata.org/wiki/Q355989","display_name":"Adeno-associated virus","level":5,"score":0.9004179239273071},{"id":"https://openalex.org/C2775905019","wikidata":"https://www.wikidata.org/wiki/Q192572","display_name":"In silico","level":3,"score":0.8946611881256104},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.642215371131897},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5460472702980042},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.5094757676124573},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.4737998843193054},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.40451180934906006},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.38163089752197266},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.06997567415237427},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.0}],"mesh":[],"locations_count":1,"locations":[{"id":"mag:2743983049","doi":null,"is_oa":true,"landing_page_url":"https://iovs.arvojournals.org/article.aspx?articleid=2641228","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":true,"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":null,"is_accepted":false,"is_published":false,"raw_source_name":"Investigative Ophthalmology & Visual Science","raw_type":null}],"best_oa_location":{"id":"mag:2743983049","doi":null,"is_oa":true,"landing_page_url":"https://iovs.arvojournals.org/article.aspx?articleid=2641228","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":true,"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":null,"is_accepted":false,"is_published":false,"raw_source_name":"Investigative Ophthalmology & Visual Science","raw_type":null},"sustainable_development_goals":[{"display_name":"Good health and well-being","score":0.9391000270843506,"id":"https://metadata.un.org/sdg/3"}],"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/W4238830363","https://openalex.org/W1197841791","https://openalex.org/W827898145","https://openalex.org/W826358037","https://openalex.org/W2998788082","https://openalex.org/W2576496914","https://openalex.org/W2528392394","https://openalex.org/W858116547","https://openalex.org/W947758719","https://openalex.org/W1189913306"],"abstract_inverted_index":null,"counts_by_year":[{"year":2023,"cited_by_count":1}],"updated_date":"2026-10-28T10:33:24.094632","created_date":"2017-08-17T00:00:00"}