PMID- 33072782 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20201020 IS - 2296-858X (Print) IS - 2296-858X (Electronic) IS - 2296-858X (Linking) VI - 7 DP - 2020 TI - Possible Novel Therapeutic Targets in Lymphangioleiomyomatosis Treatment. PG - 554134 LID - 10.3389/fmed.2020.554134 [doi] LID - 554134 AB - Lymphangioleiomyomatosis (LAM) is a rare systemic neoplastic disease that exclusively happens in women. Studies focusing on LAM and tuberous sclerosis complex (TSC) have made great progress in understanding the pathogenesis and searching for treatment. The inactive mutation of TSC1 or TSC2 is found in patients with LAM to activate the crucial mammalian target of rapamycin (mTOR) signaling pathway and result in enhanced cell proliferation and migration. However, it does not explain every step of tumorigenesis in LAM. Because cessation of rapamycin would break the stabilization of lung function or improved quality of life and lead to disease recurrent, continued studies on the pathogenesis of LAM are necessary to identify novel targets and new treatment. Researchers have found several aberrant regulations that affect the mTOR pathway such as its upstream or downstream molecules and compensatory pathways in LAM. Some therapeutic targets have been under study in clinical trials. New methods like genome-wide association studies have located a novel gene related to LAM. Herein, we review the current knowledge regarding pathogenesis and treatment of LAM and summarize novel targets of therapeutic potential recently. CI - Copyright (c) 2020 Song, Cai, Yang, Xue, Wang, Mo, Zhu, Zhu, Ye and Jin. FAU - Song, Xixi AU - Song X AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Cai, Hui AU - Cai H AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Yang, Chengyu AU - Yang C AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Xue, Xiaomin AU - Xue X AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Wang, Jian AU - Wang J AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Mo, Yuqing AU - Mo Y AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Zhu, Mengchan AU - Zhu M AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Zhu, Guiping AU - Zhu G AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Ye, Ling AU - Ye L AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. FAU - Jin, Meiling AU - Jin M AD - Department of Pulmonary and Critical Care Medicine, Zhongshan Hospital, Fudan University, Shanghai, China. LA - eng PT - Journal Article PT - Review DEP - 20200924 PL - Switzerland TA - Front Med (Lausanne) JT - Frontiers in medicine JID - 101648047 PMC - PMC7542236 OTO - NOTNLM OT - lymphangioleiomyomatosis OT - mTOR OT - pathogenesis OT - target OT - treatment EDAT- 2020/10/20 06:00 MHDA- 2020/10/20 06:01 PMCR- 2020/09/24 CRDT- 2020/10/19 05:59 PHST- 2020/04/21 00:00 [received] PHST- 2020/08/13 00:00 [accepted] PHST- 2020/10/19 05:59 [entrez] PHST- 2020/10/20 06:00 [pubmed] PHST- 2020/10/20 06:01 [medline] PHST- 2020/09/24 00:00 [pmc-release] AID - 10.3389/fmed.2020.554134 [doi] PST - epublish SO - Front Med (Lausanne). 2020 Sep 24;7:554134. doi: 10.3389/fmed.2020.554134. eCollection 2020.