{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T17:37:01Z","timestamp":1784828221095,"version":"3.55.0"},"reference-count":40,"publisher":"American Society for Clinical Investigation","issue":"4","license":[{"start":{"date-parts":[[2026,2,16]],"date-time":"2026-02-16T00:00:00Z","timestamp":1771200000000},"content-version":"vor","delay-in-days":62,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["R35 CA231998"],"award-info":[{"award-number":["R35 CA231998"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["P30 CA008748"],"award-info":[{"award-number":["P30 CA008748"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["K99 HL150628"],"award-info":[{"award-number":["K99 HL150628"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["UL1 TR001866"],"award-info":[{"award-number":["UL1 TR001866"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"name":"V Foundation Translational Grant","award":["T2019-013"],"award-info":[{"award-number":["T2019-013"]}]},{"DOI":"10.13039\/100002086","name":"Fanconi Cancer Foundation","doi-asserted-by":"crossref","award":["none"],"award-info":[{"award-number":["none"]}],"id":[{"id":"10.13039\/100002086","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Stand Up To Cancer\u2013Fanconi Cancer Foundation\u2013Farrah Fawcett Foundation Head and Neck Cancer Research Team Grant","award":["none"],"award-info":[{"award-number":["none"]}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["T32GM152349"],"award-info":[{"award-number":["T32GM152349"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Dutch Cancer Foundation","award":["12797\/2019-2"],"award-info":[{"award-number":["12797\/2019-2"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,2,16]]},"abstract":"<jats:p>Fanconi anemia (FA) confers a high risk (~700-fold increase) of solid tumor formation, most often head and neck squamous cell carcinoma (HNSCC). FA germline DNA repair defects preclude administration of most chemotherapies, and prior hematopoietic stem cell transplantation limits the use of immunotherapy. Thus, surgery and judicious delivery of radiation offer the only treatment options, with most patients dying from their cancers. A paucity of preclinical models has limited the development of new treatments. Here, we report what to our knowledge are the first patient-derived xenografts (PDXs) of FA-associated HNSCC (FA-HNSCC) and highlight the efficacy of FDA-approved EGFR-targeted therapies in tumors with high EGFR and phosphorylated EGFR levels and the activity of the FDA-approved B-cell lymphoma 2 (Bcl-2) inhibitor venetoclax in a FA-HNSCC PDX overexpressing Bcl-2. These findings support the development of precision medicine approaches for FA-HNSCC.<\/jats:p>","DOI":"10.1172\/jci195334","type":"journal-article","created":{"date-parts":[[2025,12,16]],"date-time":"2025-12-16T18:00:17Z","timestamp":1765908017000},"source":"Crossref","is-referenced-by-count":1,"title":["Patient-derived xenograft models of Fanconi anemia\u2013associated head and neck cancer identify personalized therapeutic strategies"],"prefix":"10.1172","volume":"136","author":[{"given":"Jennifer R.","family":"Grandis","sequence":"first","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, UCSF, San Francisco, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hua","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, UCSF, San Francisco, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin A.","family":"Harrison","sequence":"additional","affiliation":[{"name":"Laboratory of Genome Maintenance, The Rockefeller University, New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew L.H.","family":"Webster","sequence":"additional","affiliation":[{"name":"Laboratory of Genome Maintenance, The Rockefeller University, New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna","family":"Pucilowska","sequence":"additional","affiliation":[{"name":"Immune Monitoring and Omics Lab, Knight Cancer Institute, Oregon Health Sciences University, Portland, Oregon, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Austin","family":"Nguyen","sequence":"additional","affiliation":[{"name":"Immune Monitoring and Omics Lab, Knight Cancer Institute, Oregon Health Sciences University, Portland, Oregon, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinho","family":"Lee","sequence":"additional","affiliation":[{"name":"Immune Monitoring and Omics Lab, Knight Cancer Institute, Oregon Health Sciences University, Portland, Oregon, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0144-9614","authenticated-orcid":true,"given":"Gordon B.","family":"Mills","sequence":"additional","affiliation":[{"name":"Immune Monitoring and Omics Lab, Knight Cancer Institute, Oregon Health Sciences University, Portland, Oregon, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jovanka","family":"Gencel-Augusto","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, UCSF, San Francisco, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yan","family":"Zeng","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, UCSF, San Francisco, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven R.","family":"Long","sequence":"additional","affiliation":[{"name":"Department of Pathology, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7712-3653","authenticated-orcid":true,"given":"Mi-Ok","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Epidemiology and Biostatistics, UCSF, San Francisco, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4111-9975","authenticated-orcid":true,"given":"Rex H.","family":"Lee","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, UCSF, San Francisco, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David I.","family":"Kutler","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6200-0231","authenticated-orcid":true,"given":"Theresa","family":"Scognamiglio","sequence":"additional","affiliation":[{"name":"Department of Pathology, Weill Cornell Medical College, New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margaret","family":"Brandwein-Weber","sequence":"additional","affiliation":[{"name":"Department of Pathology, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Urken","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, Icahn School of Medicine at Mt. Sinai, New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Inna","family":"Khodos","sequence":"additional","affiliation":[{"name":"Antitumor Assessment Core, Memorial Sloan Kettering Cancer Center (MSKCC), New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3873-315X","authenticated-orcid":true,"given":"Elisa","family":"de Stanchina","sequence":"additional","affiliation":[{"name":"Antitumor Assessment Core, Memorial Sloan Kettering Cancer Center (MSKCC), New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yu-Chien","family":"Lin","sequence":"additional","affiliation":[{"name":"Laboratory of Genome Maintenance, The Rockefeller University, New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank X.","family":"Donovan","sequence":"additional","affiliation":[{"name":"National Human Genome Research Institute, NIH, Bethesda, Maryland, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Settara C.","family":"Chandrasekharappa","sequence":"additional","affiliation":[{"name":"National Human Genome Research Institute, NIH, Bethesda, Maryland, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1364-1814","authenticated-orcid":true,"given":"Moonjung","family":"Jung","sequence":"additional","affiliation":[{"name":"Laboratory of Genome Maintenance, The Rockefeller University, New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mathijs A.","family":"Sanders","sequence":"additional","affiliation":[{"name":"Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6285-1562","authenticated-orcid":true,"given":"Agata","family":"Smogorzewska","sequence":"additional","affiliation":[{"name":"Laboratory of Genome Maintenance, The Rockefeller University, New York, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2505-530X","authenticated-orcid":true,"given":"Daniel E.","family":"Johnson","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology \u2013 Head and Neck Surgery, UCSF, San Francisco, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"232","published-online":{"date-parts":[[2025,12,16]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI185126"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI187323"},{"key":"B3","unstructured":"Fanconi Cancer Foundation. 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