Article

Genome-wide functional genomics screens identify therapeutic vulnerabilities for lymphangioleiomyomatosis

2019-06-27

Abstract excerpt

Lymphangioleiomyomatosis (LAM) is a rare lung disease marked by cystic destruction caused by invasive LAM cells harboring loss-of-function mutations in TSC2 that exhibit dysregulated mTORC1 signaling. Rapamycin, the only approved treatment, is not curative. Therapeutic discovery has been limited by inadequate cell and animal models due to the unknown LAM cell of origin. We report a novel TSC2 —/— human pluripot...

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Literature Corpus work: 6a795ebf-2c3a-57e0-80e8-7d9d32ad9227
DOI: 10.1101/683359

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