{"id":"https://openalex.org/W4386705487","doi":null,"title":"THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE.","display_name":"THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE.","publication_year":2023,"publication_date":"2023-01-01","ids":{"openalex":"https://openalex.org/W4386705487","pmid":"https://pubmed.ncbi.nlm.nih.gov/37701622","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/10493758"},"language":"en","primary_location":{"id":"pmh:oai:pubmedcentral.nih.gov:10493758","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/10493758","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC10493758/pdf/tacca1330000204.pdf","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":"Trans Am Clin Climatol Assoc","raw_type":"Text"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC10493758/pdf/tacca1330000204.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5060779925","display_name":"Katherine A. High","orcid":"https://orcid.org/0000-0002-0805-6223","observed_orcids":["https://orcid.org/0000-0002-0805-6223"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Katherine A High","raw_affiliation_strings":["New York, New York"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"New York, New York","institution_ids":[]}]}],"institutions":[],"countries_distinct_count":0,"institutions_distinct_count":0,"corresponding_author_ids":["https://openalex.org/A5060779925"],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":0.0,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":{"value":0.0,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"133","issue":null,"first_page":"204","last_page":"233"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9735999703407288,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9735999703407288,"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":0.004800000227987766,"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/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.003800000064074993,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"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/gene-therapy","display_name":"gene therapy","score":0.9869999885559082},{"id":"https://openalex.org/keywords/genetic-disorders","display_name":"genetic disorders","score":0.796999990940094},{"id":"https://openalex.org/keywords/clinical-drug-development","display_name":"clinical development","score":0.35899999737739563},{"id":"https://openalex.org/keywords/clinical-trial-endpoints","display_name":"clinical trial endpoints","score":0.7919999957084656},{"id":"https://openalex.org/keywords/drug-therapy","display_name":"pharmacotherapy","score":0.5519999861717224}],"concepts":[{"id":"https://openalex.org/C2780724529","wikidata":"https://www.wikidata.org/wiki/Q18031223","display_name":"RPE65","level":4,"score":0.8087825179100037},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.7637706995010376},{"id":"https://openalex.org/C3018928802","wikidata":"https://www.wikidata.org/wiki/Q83185","display_name":"Gene transfer","level":3,"score":0.5656476616859436},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.5485738515853882},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.5301694869995117},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.5119086503982544},{"id":"https://openalex.org/C144501496","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome editing","level":4,"score":0.49724510312080383},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.4938269555568695},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.4459458589553833},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.39316272735595703},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.37548908591270447},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.36061352491378784},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.3519189953804016},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.33190298080444336},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.2206411063671112},{"id":"https://openalex.org/C141231307","wikidata":"https://www.wikidata.org/wiki/Q7020","display_name":"Genome","level":3,"score":0.1329435408115387},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.09331881999969482}],"mesh":[{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002836","descriptor_name":"Hemophilia B","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D002836","descriptor_name":"Hemophilia B","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008322","descriptor_name":"Mammals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009154","descriptor_name":"Mutation","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":false}],"locations_count":2,"locations":[{"id":"pmh:oai:pubmedcentral.nih.gov:10493758","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/10493758","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC10493758/pdf/tacca1330000204.pdf","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":"Trans Am Clin Climatol Assoc","raw_type":"Text"},{"id":"pmid:37701622","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/37701622","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":"Transactions of the American Clinical and Climatological Association","raw_type":"Journal Article"}],"best_oa_location":{"id":"pmh:oai:pubmedcentral.nih.gov:10493758","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/10493758","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC10493758/pdf/tacca1330000204.pdf","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":"Trans Am Clin Climatol Assoc","raw_type":"Text"},"sustainable_development_goals":[{"score":0.9391000270843506,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4386705487.pdf","grobid_xml":"https://content.openalex.org/works/W4386705487.grobid-xml"},"referenced_works_count":57,"referenced_works":["https://openalex.org/W1487084354","https://openalex.org/W1511807972","https://openalex.org/W1922286450","https://openalex.org/W1968382713","https://openalex.org/W1969271703","https://openalex.org/W1970138286","https://openalex.org/W1975528602","https://openalex.org/W1976329054","https://openalex.org/W1976452565","https://openalex.org/W1978519692","https://openalex.org/W1980332309","https://openalex.org/W1988197108","https://openalex.org/W1998165633","https://openalex.org/W2000633059","https://openalex.org/W2003970226","https://openalex.org/W2005059927","https://openalex.org/W2007261697","https://openalex.org/W2030494254","https://openalex.org/W2032678451","https://openalex.org/W2035101755","https://openalex.org/W2051140382","https://openalex.org/W2059827251","https://openalex.org/W2072373173","https://openalex.org/W2080951809","https://openalex.org/W2086698948","https://openalex.org/W2091718181","https://openalex.org/W2093424805","https://openalex.org/W2121444412","https://openalex.org/W2122304523","https://openalex.org/W2130515212","https://openalex.org/W2137222303","https://openalex.org/W2137440275","https://openalex.org/W2141354154","https://openalex.org/W2141575938","https://openalex.org/W2143729842","https://openalex.org/W2151809142","https://openalex.org/W2155710930","https://openalex.org/W2162290849","https://openalex.org/W2163889167","https://openalex.org/W2411144158","https://openalex.org/W2471671770","https://openalex.org/W2473832992","https://openalex.org/W2734428113","https://openalex.org/W2736065260","https://openalex.org/W2765253110","https://openalex.org/W2773253161","https://openalex.org/W2894534989","https://openalex.org/W3005529704","https://openalex.org/W3016405424","https://openalex.org/W3017171049","https://openalex.org/W3035047148","https://openalex.org/W3093323554","https://openalex.org/W3105402850","https://openalex.org/W3211832397","https://openalex.org/W4231146812","https://openalex.org/W4281664094","https://openalex.org/W4321480301"],"related_works":["https://openalex.org/W2019013094","https://openalex.org/W2402678354","https://openalex.org/W7052906035","https://openalex.org/W2275939941","https://openalex.org/W2414261112","https://openalex.org/W2409746860","https://openalex.org/W2143882113","https://openalex.org/W962070469","https://openalex.org/W4408350523","https://openalex.org/W2121527848"],"abstract_inverted_index":{"deficiency,":[0],"a":[1,10,15,20],"key":[2],"to":[3],"success":[4],"was":[5],"development":[6],"and":[7],"validation":[8],"of":[9],"novel":[11],"clinical":[12],"endpoint":[13],"for":[14],"disease":[16],"that":[17],"previously":[18],"lacked":[19],"pharmacologic":[21],"treatment.":[22]},"counts_by_year":[],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2023-09-14T00:00:00"}