Article

Three-dimensional drug screen identifies HDAC inhibitors as therapeutic agents in mTORC1 <i>-</i> driven lymphangioleiomyomatosis

2021-07-04

Abstract excerpt

<h4>ABSTRACT</h4> Lymphangioleiomyomatosis (LAM) is a rare disease involving cystic lung destruction by invasive LAM cells. These cells harbor loss-of-function mutations in TSC2 , conferring constitutive mTORC1 signaling. Rapamycin is the only clinically approved disease-modifying treatment, but its action is cytostatic and disease progresses upon its withdrawal. There is a critical need to identify novel agents...

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Literature Corpus work: 548bef31-1325-5bdc-96ec-86c116951bfd
DOI: 10.1101/2021.07.03.451004

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