神经纤维瘤病（NF1）是一种常见的显性遗传疾病，也是拉索病的常见类型之一。大多数患有NF1的个体会发展出网状神经纤维瘤和皮肤神经纤维瘤，这是由于施万细胞中NF1功能丧失导致的神经肿瘤。细胞培养模型和NF1小鼠模型正在用于测试临床前试验中的药物功效，这导致了美国食品药品监督管理局批准使用MEK抑制剂来减小大多数不可手术的网状神经纤维瘤。本文详细介绍了用于临床前模型测试的方法，并概述了可能发现额外治愈策略的更新模型。版权所有 © 2023 Elsevier Inc. 保留所有权利。

Neurofibromatosis type I (NF1) is a common dominantly inherited disorder, and one of the most common of the RASopathies. Most individuals with NF1 develop plexiform neurofibromas and cutaneous neurofibromas, nerve tumors caused by NF1 loss of function in Schwann cells. Cell culture models and mouse models of NF1 are being used to test drug efficacy in preclinical trials, which led to Food and Drug Administration approval for use of MEK inhibitors to shrink most inoperable plexiform neurofibromas. This article details methods used for testing in preclinical models, and outlines newer models that may identify additional, curative, strategies.Copyright © 2023 Elsevier Inc. All rights reserved.