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Neurofibromatosis Type 1 and the Search for Effective Tumor Therapies Using High-Throughput Drug Screening.

Created on 14 Oct 2026

Authors

Stephanie J Bouley, Benjamin E Housden, James A Walker

Published in

Current oncology (Toronto, Ont.). Volume 32. Issue 11. Nov 20, 2025. Epub Nov 20, 2025.

Abstract

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.

PMID:
41294711
Bibliographic data and abstract were imported from PubMed on 14 Oct 2026.

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