{"id":"https://openalex.org/W4367305162","doi":"https://doi.org/10.1212/wnl.0000000000202663","title":"Comparison of Therapeutic Efficacy of Gene Therapy for Tuberous Sclerosis Type 2 with Standard of Care Everolimus in Preclinical Model (S14.002)","display_name":"Comparison of Therapeutic Efficacy of Gene Therapy for Tuberous Sclerosis Type 2 with Standard of Care Everolimus in Preclinical Model (S14.002)","publication_year":2023,"publication_date":"2023-04-25","ids":{"openalex":"https://openalex.org/W4367305162","doi":"https://doi.org/10.1212/wnl.0000000000202663"},"language":"en","primary_location":{"id":"doi:10.1212/wnl.0000000000202663","doi":"https://doi.org/10.1212/wnl.0000000000202663","is_oa":false,"landing_page_url":"https://doi.org/10.1212/wnl.0000000000202663","pdf_url":null,"source":{"id":"https://openalex.org/S79704628","display_name":"Neurology","issn_l":"0028-3878","issn":["0028-3878","1526-632X"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310315671","host_organization_name":"Lippincott Williams & Wilkins","host_organization_lineage":["https://openalex.org/P4310315671","https://openalex.org/P4310318547"],"host_organization_lineage_names":["Lippincott Williams & Wilkins","Wolters Kluwer"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Neurology","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref"],"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/A5016545930","display_name":"Edwina Abou Haidar","orcid":"https://orcid.org/0000-0002-7866-8030","observed_orcids":["https://orcid.org/0000-0002-7866-8030"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Edwina Abou Haidar","raw_affiliation_strings":["Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5045740999","display_name":"Shilpa Prabhakar","orcid":"https://orcid.org/0000-0002-7999-2000","observed_orcids":["https://orcid.org/0000-0002-7999-2000"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Shilpa Prabhakar","raw_affiliation_strings":["Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5015044138","display_name":"Pike-See Cheah","orcid":"https://orcid.org/0000-0003-2181-2502","observed_orcids":["https://orcid.org/0000-0003-2181-2502"]},"institutions":[{"id":"https://openalex.org/I130343225","display_name":"Universiti Putra Malaysia","ror":"https://ror.org/02e91jd64","country_code":"MY","type":"education","lineage":["https://openalex.org/I130343225"]}],"countries":["MY"],"is_corresponding":false,"raw_author_name":"Pike See Cheah","raw_affiliation_strings":["Department of Human Anatomy, Faculty of Medicine and Health Sciences, Universiti Putra Malaysia, Malaysia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Human Anatomy, Faculty of Medicine and Health Sciences, Universiti Putra Malaysia, Malaysia","institution_ids":["https://openalex.org/I130343225"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5000816967","display_name":"Sevda L\u00fcle","orcid":"https://orcid.org/0000-0001-7066-6471","observed_orcids":["https://orcid.org/0000-0001-7066-6471"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Sevda Lule","raw_affiliation_strings":["Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5032940778","display_name":"Roberta L. Beauchamp","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Roberta L. Beauchamp","raw_affiliation_strings":["Center for Human Genetics Research, MGH, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Human Genetics Research, MGH, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5016860642","display_name":"Akiko Yoshinaga","orcid":"https://orcid.org/0009-0002-2159-1164","observed_orcids":["https://orcid.org/0009-0002-2159-1164"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Akiko Yoshinaga","raw_affiliation_strings":["Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5031700387","display_name":"Alexandra L. Geffrey","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Alexandra L. Geffrey","raw_affiliation_strings":["Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5091816977","display_name":"Anat O Stemmer-Rachamimov","orcid":"https://orcid.org/0000-0003-3846-5295","observed_orcids":["https://orcid.org/0000-0003-3846-5295"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Anat Stemmer-Rachamimov","raw_affiliation_strings":["Department of Pathology, Boston, MA USA, Massachusetts General Hospital"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pathology, Boston, MA USA, Massachusetts General Hospital","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5011818435","display_name":"Vijaya Ramesh","orcid":"https://orcid.org/0000-0003-4966-5268","observed_orcids":["https://orcid.org/0000-0003-4966-5268"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Vijaya Ramesh","raw_affiliation_strings":["Center for Human Genetics Research, MGH, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Human Genetics Research, MGH, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5064371575","display_name":"Casey A. Maguire","orcid":"https://orcid.org/0000-0001-8681-5179","observed_orcids":["https://orcid.org/0000-0001-8681-5179"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Casey A. Maguire","raw_affiliation_strings":["Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5059573074","display_name":"Xandra O. Breakefield","orcid":"https://orcid.org/0000-0001-6036-0399","observed_orcids":["https://orcid.org/0000-0001-6036-0399"]},"institutions":[{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Xandra O. Breakefield","raw_affiliation_strings":["Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, MGH, and Program in Neuroscience, HMS, Boston, MA USA","institution_ids":["https://openalex.org/I4210087915"]}]}],"institutions":[],"countries_distinct_count":2,"institutions_distinct_count":2,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":0.0,"has_fulltext":false,"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":"100","issue":"17_supplement_2","first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T12049","display_name":"Tuberous Sclerosis Complex Research","score":0.9970999956130981,"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"}},"topics":[{"id":"https://openalex.org/T12049","display_name":"Tuberous Sclerosis Complex Research","score":0.9970999956130981,"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"}},{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.002099999925121665,"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":9.999999747378752e-05,"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":0.8960000276565552},{"id":"https://openalex.org/keywords/adeno-associated-virus-vectors","display_name":"adeno-associated virus vectors","score":0.859000027179718},{"id":"https://openalex.org/keywords/everolimus","display_name":"everolimus","score":0.9309999942779541},{"id":"https://openalex.org/keywords/brain-lesions","display_name":"brain lesions","score":0.8479999899864197},{"id":"https://openalex.org/keywords/treatment-efficacy","display_name":"treatment efficacy","score":0.6949999928474426}],"concepts":[{"id":"https://openalex.org/C2779422105","wikidata":"https://www.wikidata.org/wiki/Q21109261","display_name":"TSC2","level":4,"score":0.9663739204406738},{"id":"https://openalex.org/C2778980267","wikidata":"https://www.wikidata.org/wiki/Q1362721","display_name":"Tuberous sclerosis","level":2,"score":0.9391215443611145},{"id":"https://openalex.org/C2776773979","wikidata":"https://www.wikidata.org/wiki/Q898669","display_name":"TSC1","level":4,"score":0.9026528596878052},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.5906442999839783},{"id":"https://openalex.org/C2779699572","wikidata":"https://www.wikidata.org/wiki/Q421052","display_name":"Everolimus","level":2,"score":0.5565890669822693},{"id":"https://openalex.org/C86554907","wikidata":"https://www.wikidata.org/wiki/Q285613","display_name":"PI3K/AKT/mTOR pathway","level":3,"score":0.5162723064422607},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.486131876707077},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.46092885732650757},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.3733600378036499},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.34692496061325073},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.2919723391532898},{"id":"https://openalex.org/C62478195","wikidata":"https://www.wikidata.org/wiki/Q828130","display_name":"Signal transduction","level":2,"score":0.1887054741382599},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.17799121141433716},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.1487172245979309}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.1212/wnl.0000000000202663","doi":"https://doi.org/10.1212/wnl.0000000000202663","is_oa":false,"landing_page_url":"https://doi.org/10.1212/wnl.0000000000202663","pdf_url":null,"source":{"id":"https://openalex.org/S79704628","display_name":"Neurology","issn_l":"0028-3878","issn":["0028-3878","1526-632X"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310315671","host_organization_name":"Lippincott Williams & Wilkins","host_organization_lineage":["https://openalex.org/P4310315671","https://openalex.org/P4310318547"],"host_organization_lineage_names":["Lippincott Williams & Wilkins","Wolters Kluwer"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Neurology","raw_type":"journal-article"}],"best_oa_location":null,"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","score":0.9940000176429749,"display_name":"Good health and well-being"}],"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/W330343617","https://openalex.org/W3119625315","https://openalex.org/W2563770260","https://openalex.org/W2156256589","https://openalex.org/W4413163068","https://openalex.org/W3120274187","https://openalex.org/W3004653360","https://openalex.org/W7135907598","https://openalex.org/W45388609","https://openalex.org/W7164896960"],"abstract_inverted_index":{"Therapeutic":[0],"efficacy":[1],"of":[2],"gene":[3],"therapy":[4],"using":[5],"an":[6],"adeno-associated":[7],"viral":[8],"vector":[9],"encoding":[10],"cTuberin":[11],"to":[12],"treat":[13],"brain":[14],"lesions":[15],"in":[16],"a":[17],"TSC2":[18],"mouse":[19],"model.":[20]},"counts_by_year":[],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2023-04-29T00:00:00"}