Extract
Lymphangioleiomyomatosis (LAM) is a slowly progressive, low-grade neoplasm predominantly affecting women that originates from tuberous sclerosis complex (TSC)-deficient cells of mesenchymal lineage (“LAM cells”) in the uterus or pelvis [1, 2]. These LAM cells metastasise through the lymphatics to the abdominal and retroperitoneal lymph nodes, kidneys and lung [1–5]. LAM cells have loss-of-function mutations in both alleles of eitherTSC1或者TSC2该功能抑制雷帕霉素复合物1(MTORC1)的机械靶标。在没有补品抑制的情况下,MTORC1途径是活跃的,可以驱动LAM细胞的增殖和侵袭。抑制MTORC1的西罗莫司(雷帕霉素)和依维洛木斯(Everolimus)等药物抑制了LAM细胞的生长和侵入性,并且在减慢LAM疾病的进展方面在临床上有效[6-8],尽管这种作用是可逆的,因此需要继续治疗。尽管治疗和/或无法忍受药物,但仍有一部分患者进展。目前,除了监测肺功能测试[9],血清血管内皮生长因子D [10]和计算机断层扫描的囊肿进展之外,所有这些都是显微镜疾病进展的滞后指标,没有临床上有用的方法监测疾病活动/进展和对治疗的反应。
Abstract
Use of proteinase-specific nanosensors holds great promise to measure lymphangioleiomyomatosis lung disease activity and response to treatmenthttps://bit.ly/3uEsVhF
脚注
利益冲突:G.P。Downey报告了NIH的赠款,Angion Biomedica和Roche/Genentech的咨询费;是美国胸部学会总统(向机构支付)。Y. Aschner报告了NIH的赠款。
Support statement: Supported by funding from the National Institutes of Health (HL132950 and HL157424 to G.P. Downey), NIH/NCATS Colorado CTSA (KL2 TR002534 and K08HL135279-01A1 to Y. Aschner), and the Parker B. Francis Foundation (Y. Aschner). Funding information for this article has been deposited with theCrossref Funder Registry.
- 已收到February 23, 2022.
- AcceptedMarch 17, 2022.
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