An shRNA based screening method for TSC synthetic lethal screens

PI: Norbert Perrimon, PhD

Institution: Harvard Medical School


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Lymphangioleiomyomatosis (LAM) is a rare lung disease mostly affecting women. The disease is associated with the uncontrolled growth of cells, particularly in lungs, lymph nodes, and kidneys. The disease is associated with mutations in two genes, TSC1 and TSC2. Current therapeutic strategies targeting TSC1 and TSC2 are limited to cytostatic effects and tumors rapidly regrow upon cessation of treatment. Thus, there is a great need to discover new drug targets that can be used to treat TSC. To discover new therapeutic treatments for this disease, we are searching for genes which when mutated specifically kill TSC mutant cells but have no effect on wildtype tissue. We have developed a new screening method that we believe will identify such drug targets. To date, a number of promising candidates have been identified that are being followed actively to validate their potential uses.