{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,22]],"date-time":"2025-11-22T05:33:51Z","timestamp":1763789631328,"version":"3.45.0"},"reference-count":171,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2025,11,20]],"date-time":"2025-11-20T00:00:00Z","timestamp":1763596800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100016618","name":"Gilbert Family Foundation","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100016618","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100007500","name":"Bloomberg Family Foundation","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100007500","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000090","name":"DOD CDMRP NFRP EIRA","doi-asserted-by":"publisher","award":["HT9425-23-1-0831"],"award-info":[{"award-number":["HT9425-23-1-0831"]}],"id":[{"id":"10.13039\/100000090","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Medical Research Council"},{"DOI":"10.13039\/501100000317","name":"Action Medical Research","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100000317","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100012357","name":"LifeArc","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100012357","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Neurofibromatosis Northeast"},{"name":"CureNFwithJack"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Current Oncology"],"abstract":"<jats:p>Neurofibromatosis type 1 (NF1) is a complex, multisystem, genetic disorder caused by germline NF1 variants that predispose affected individuals to tumors of the nervous system. With the identification of the NF1 gene in the late 1980s and the elucidation of the role of the encoded protein, neurofibromin, in regulating RAS signaling, considerable research effort has been invested to identify therapeutic treatments for NF1 tumors. Over the past two decades, high-throughput drug screening approaches have been a significant component of these endeavors. However, considerable variability exists among studies in terms of disease models, symptom targets, screening libraries, methods, and outcomes. In this review, we present an overall summary of efforts toward discovering new therapeutic strategies for NF1-related tumors using high-throughput screening and how such findings can be employed for prospective research in the NF1 field.<\/jats:p>","DOI":"10.3390\/curroncol32110649","type":"journal-article","created":{"date-parts":[[2025,11,20]],"date-time":"2025-11-20T12:02:42Z","timestamp":1763640162000},"page":"649","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Neurofibromatosis Type 1 and the Search for Effective Tumor Therapies Using High-Throughput Drug Screening"],"prefix":"10.3390","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0785-0860","authenticated-orcid":false,"given":"Stephanie J.","family":"Bouley","sequence":"first","affiliation":[{"name":"Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9134-4279","authenticated-orcid":false,"given":"Benjamin E.","family":"Housden","sequence":"additional","affiliation":[{"name":"Living Systems Institute, University of Exeter, Exeter EX4 4QD, UK"},{"name":"Department of Clinical and Biomedical Science, University of Exeter, Exeter EX4 4QJ, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5585-698X","authenticated-orcid":false,"given":"James A.","family":"Walker","sequence":"additional","affiliation":[{"name":"Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA"},{"name":"Cancer Program, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA"},{"name":"Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2025,11,20]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"292","DOI":"10.1186\/s13023-023-02911-2","article-title":"Incidence and Prevalence of Neurofibromatosis Type 1 and 2: A Systematic Review and Meta-Analysis","volume":"18","author":"Lee","year":"2023","journal-title":"Orphanet J. 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