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

Current oncology (Toronto, Ont.)
Authors
Keywords
Abstract

Neurofibromatosis type 1 (NF1) is a complex, multisystem, genetic disorder caused by germline variants that predispose affected individuals to tumors of the nervous system. With the identification of the 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.

Year of Publication
2025
Journal
Current oncology (Toronto, Ont.)
Volume
32
Issue
11
Date Published
11/2025
ISSN
1718-7729
DOI
10.3390/curroncol32110649
PubMed ID
41294711
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