{"id":"https://openalex.org/W6989021971","doi":null,"title":"Advancing Gene Therapy Technologies for the Treatment of Genetic Disorders","display_name":"Advancing Gene Therapy Technologies for the Treatment of Genetic Disorders","publication_year":2023,"publication_date":"2023-01-01","ids":{"openalex":"https://openalex.org/W6989021971"},"language":"en","primary_location":{"id":"pmh:oai:escholarship.org:ark:/13030/qt1rj6t7v7","doi":null,"is_oa":true,"landing_page_url":"https://escholarship.org/uc/item/1rj6t7v7","pdf_url":"https://escholarship.org/content/qt1rj6t7v7/qt1rj6t7v7.pdf","source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"","raw_type":"etd"},"volume":null,"type":"dissertation","is_traditional":true,"indexed_in":[],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://escholarship.org/content/qt1rj6t7v7/qt1rj6t7v7.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5061737233","display_name":"Paul George Ayoub","orcid":"https://orcid.org/0000-0003-3050-2397","observed_orcids":["https://orcid.org/0000-0003-3050-2397"]},"institutions":[{"id":"https://openalex.org/I161318765","display_name":"University of California, Los Angeles","ror":"https://ror.org/046rm7j60","country_code":"US","type":"education","lineage":["https://openalex.org/I161318765"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Ayoub, Paul George","raw_affiliation_strings":["Doctor of Philosophy in Molecular and Medical Pharmacology University of California , Los Angeles , 2023"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Doctor of Philosophy in Molecular and Medical Pharmacology University of California , Los Angeles , 2023","institution_ids":["https://openalex.org/I161318765"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5061737233"],"corresponding_institution_ids":["https://openalex.org/I161318765"],"apc_list":null,"apc_paid":null,"fwci":null,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":true,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9980000257492065,"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":0.9980000257492065,"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/T10878","display_name":"CRISPR and Genetic Engineering","score":0.0015999999595806003,"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/T10725","display_name":"RNA Interference and Gene Delivery","score":0.0003000000142492354,"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":0.9739999771118164},{"id":"https://openalex.org/keywords/genetic-disorders","display_name":"genetic disorders","score":0.8519999980926514},{"id":"https://openalex.org/keywords/lentiviral-vectors","display_name":"lentiviral vectors","score":0.9869999885559082},{"id":"https://openalex.org/keywords/x-linked-lymphoproliferative-disease","display_name":"X-linked lymphoproliferative disease","score":0.8730000257492065},{"id":"https://openalex.org/keywords/genome-editing","display_name":"genome editing","score":0.6000000238418579},{"id":"https://openalex.org/keywords/cystic-fibrosis","display_name":"cystic fibrosis","score":0.996999979019165},{"id":"https://openalex.org/keywords/small-molecule-inhibitors","display_name":"small molecule inhibitors","score":0.7590000033378601}],"concepts":[{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.7432000041007996},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.6452000141143799},{"id":"https://openalex.org/C186852380","wikidata":"https://www.wikidata.org/wiki/Q1142521","display_name":"Electroporation","level":3,"score":0.6448000073432922},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.6047000288963318},{"id":"https://openalex.org/C144501496","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome editing","level":4,"score":0.5945000052452087},{"id":"https://openalex.org/C15152581","wikidata":"https://www.wikidata.org/wiki/Q7833966","display_name":"Transduction (biophysics)","level":2,"score":0.5788999795913696},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.5271999835968018},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5115000009536743},{"id":"https://openalex.org/C135983454","wikidata":"https://www.wikidata.org/wiki/Q2790163","display_name":"Gene delivery","level":4,"score":0.4934999942779541},{"id":"https://openalex.org/C111829913","wikidata":"https://www.wikidata.org/wiki/Q211083","display_name":"Gene targeting","level":3,"score":0.41760000586509705},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.40950000286102295},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.3522999882698059},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.3287000060081482},{"id":"https://openalex.org/C43953973","wikidata":"https://www.wikidata.org/wiki/Q7917858","display_name":"Vectors in gene therapy","level":5,"score":0.31610000133514404},{"id":"https://openalex.org/C35802184","wikidata":"https://www.wikidata.org/wiki/Q204773","display_name":"Zinc finger nuclease","level":5,"score":0.2921000123023987},{"id":"https://openalex.org/C552990157","wikidata":"https://www.wikidata.org/wiki/Q7430","display_name":"DNA","level":2,"score":0.2904999852180481},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.27900001406669617},{"id":"https://openalex.org/C51679486","wikidata":"https://www.wikidata.org/wiki/Q380546","display_name":"DNA sequencing","level":3,"score":0.2678000032901764},{"id":"https://openalex.org/C187882448","wikidata":"https://www.wikidata.org/wiki/Q283478","display_name":"Complementary DNA","level":3,"score":0.2676999866962433},{"id":"https://openalex.org/C41008148","wikidata":"https://www.wikidata.org/wiki/Q21198","display_name":"Computer science","level":0,"score":0.2671000063419342},{"id":"https://openalex.org/C2776904630","wikidata":"https://www.wikidata.org/wiki/Q356336","display_name":"Adept","level":3,"score":0.2639000117778778},{"id":"https://openalex.org/C16613235","wikidata":"https://www.wikidata.org/wiki/Q285982","display_name":"Endogeny","level":2,"score":0.2549000084400177}],"mesh":[],"locations_count":1,"locations":[{"id":"pmh:oai:escholarship.org:ark:/13030/qt1rj6t7v7","doi":null,"is_oa":true,"landing_page_url":"https://escholarship.org/uc/item/1rj6t7v7","pdf_url":"https://escholarship.org/content/qt1rj6t7v7/qt1rj6t7v7.pdf","source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"","raw_type":"etd"}],"best_oa_location":{"id":"pmh:oai:escholarship.org:ark:/13030/qt1rj6t7v7","doi":null,"is_oa":true,"landing_page_url":"https://escholarship.org/uc/item/1rj6t7v7","pdf_url":"https://escholarship.org/content/qt1rj6t7v7/qt1rj6t7v7.pdf","source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"","raw_type":"etd"},"sustainable_development_goals":[{"score":0.9476000070571899,"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/W6989021971.pdf","grobid_xml":"https://content.openalex.org/works/W6989021971.grobid-xml"},"referenced_works_count":91,"referenced_works":["https://openalex.org/W1482523072","https://openalex.org/W1487843912","https://openalex.org/W1510178802","https://openalex.org/W1966417933","https://openalex.org/W1973910785","https://openalex.org/W1975583311","https://openalex.org/W1984295407","https://openalex.org/W1989570444","https://openalex.org/W1990297263","https://openalex.org/W1990991203","https://openalex.org/W1991818026","https://openalex.org/W1992294822","https://openalex.org/W1996976023","https://openalex.org/W2009761723","https://openalex.org/W2014686737","https://openalex.org/W2024266527","https://openalex.org/W2024446703","https://openalex.org/W2027740034","https://openalex.org/W2030437588","https://openalex.org/W2039943899","https://openalex.org/W2055123567","https://openalex.org/W2063193285","https://openalex.org/W2064543601","https://openalex.org/W2065341945","https://openalex.org/W2067753430","https://openalex.org/W2071761673","https://openalex.org/W2072128608","https://openalex.org/W2076370048","https://openalex.org/W2078745581","https://openalex.org/W2079650531","https://openalex.org/W2082922487","https://openalex.org/W2088257569","https://openalex.org/W2093086410","https://openalex.org/W2112936117","https://openalex.org/W2112946827","https://openalex.org/W2118890112","https://openalex.org/W2119704076","https://openalex.org/W2131810356","https://openalex.org/W2154853638","https://openalex.org/W2156423386","https://openalex.org/W2167523976","https://openalex.org/W2168036974","https://openalex.org/W2170419443","https://openalex.org/W2203501604","https://openalex.org/W2604331373","https://openalex.org/W2606675097","https://openalex.org/W2744599076","https://openalex.org/W2790011611","https://openalex.org/W2801911828","https://openalex.org/W2909772640","https://openalex.org/W2909946034","https://openalex.org/W2968358239","https://openalex.org/W2969525890","https://openalex.org/W2975976021","https://openalex.org/W3010955108","https://openalex.org/W3013093478","https://openalex.org/W3033407907","https://openalex.org/W3039409489","https://openalex.org/W3044321933","https://openalex.org/W3044702419","https://openalex.org/W3048571801","https://openalex.org/W3058836816","https://openalex.org/W3084653951","https://openalex.org/W3088823112","https://openalex.org/W3093038587","https://openalex.org/W3096967708","https://openalex.org/W3097451149","https://openalex.org/W3099830670","https://openalex.org/W3100064532","https://openalex.org/W3109958337","https://openalex.org/W3111057263","https://openalex.org/W3111331330","https://openalex.org/W3132217836","https://openalex.org/W3133794453","https://openalex.org/W3134987603","https://openalex.org/W3153616697","https://openalex.org/W3162626720","https://openalex.org/W3189178471","https://openalex.org/W3215268416","https://openalex.org/W4206155200","https://openalex.org/W4213172983","https://openalex.org/W4223949209","https://openalex.org/W4281296218","https://openalex.org/W4293105141","https://openalex.org/W4293374782","https://openalex.org/W4307386237","https://openalex.org/W4308255675","https://openalex.org/W4385776034","https://openalex.org/W4386168613","https://openalex.org/W4386593133","https://openalex.org/W4387776286"],"related_works":["https://openalex.org/W7155680198","https://openalex.org/W2097052308","https://openalex.org/W2140033632","https://openalex.org/W3016339313","https://openalex.org/W4416733184","https://openalex.org/W4399818867","https://openalex.org/W6991444227","https://openalex.org/W3029789166","https://openalex.org/W2562501372","https://openalex.org/W4248864825"],"abstract_inverted_index":{"This":[0],"work":[1],"is":[2],"a":[3,25,58,107,118,126],"culmination":[4],"of":[5,8,16,24,29,48,57,60,91,100,104,125,133,158,165],"the":[6,14,22,55,67,83,92,101,131,141,153,163],"development":[7],"various":[9,137,145],"gene":[10,119,148,159],"therapy":[11],"technologies":[12],"for":[13,122,130],"treatment":[15,132],"genetic":[17],"disorders.":[18],"Chapter":[19,52,110],"1":[20],"highlights":[21],"generation":[23,56],"modified":[26],"S":[27],"glycoprotein":[28],"SARS-Cov-2":[30],"to":[31,89,116,147,151],"pseudotype":[32],"lentiviral":[33,61],"vectors":[34,62,77,96],"which":[35],"efficiently":[36],"transduced":[37],"ACE2-expressing":[38],"cells":[39],"with":[40],"high":[41],"specificity":[42],"and":[43,156,174],"contain":[44],"minimal":[45],"off-target":[46],"transduction":[47],"ACE2":[49],"negative":[50],"cells.":[51],"2":[53],"illustrates":[54],"series":[59],"using":[63],"regulatory":[64],"elements":[65],"from":[66],"SH2D1A":[68],"locus.":[69],"By":[70],"iterative":[71],"process,":[72],"we":[73,143],"developed":[74],"two":[75],"candidate":[76],"that":[78],"express":[79],"SAP":[80],"protein":[81],"in":[82,106],"native":[84],"pattern":[85],"at":[86],"levels":[87],"similar":[88],"those":[90],"endogenous":[93],"gene.":[94],"These":[95],"may":[97],"support":[98],"reconstitution":[99],"immunologic":[102],"deficiencies":[103],"XLP1":[105],"safe":[108],"manner.":[109],"3":[111],"delineates":[112],"an":[113],"ongoing":[114],"approach":[115],"develop":[117],"editing":[120,149,160],"strategy":[121],"site-specific":[123],"insertion":[124],"CFTR":[127],"cDNA":[128],"cassette":[129],"Cystic":[134],"Fibrosis.":[135],"Utilizing":[136],"techniques":[138],"practiced":[139],"throughout":[140],"laboratory,":[142],"designed":[144],"improvements":[146],"cargo":[150,161],"increase":[152],"cutting":[154],"efficiency":[155],"integration":[157],"including":[162],"inclusion":[164],"electroporation":[166],"enhancers,":[167],"double":[168],"stranded":[169],"DNA":[170],"donor":[171],"chemical":[172],"modifications":[173],"small":[175],"molecule":[176],"inhibitors.":[177]},"counts_by_year":[],"updated_date":"2026-11-07T11:25:07.637180","created_date":"2025-10-10T00:00:00"}