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Identifying Novel Drug Targets for the Treatment of Tuberous Sclerosis Complex Using High Throughput Technologies. Addendum.

机译:利用高通量技术鉴定新型药物靶点治疗结节性硬化症。附录。

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In a patient with Tuberous Sclerosis Complex (TSC), the problematic cells that initiate and constitute tumors have lost TSC1 or TSC2 function. A promising approach for treatment would be to target members of the pathway with which TSC1/2 proteins interact. In cultured drosophila cells, we proposed to rapidly identify genes whose RNAa-mediated reduction in expression (1) Prevents growth proliferation of TSC1 or TSC2 deficient cells without affecting normal cells. (2) induces apoptosis/cell death in TSC1 or TSC2-deficient cells without killing normal cells. (3) Reverts TSC1 or TSC2-deficient cells to a normal phenotype, as determine by measuring a reporters of cell growth pathway activation and cell morphology. We have (1) advanced genome-wide RNA interference living cell microarrays from proof-of-principle to a robust technology. (2) developed software to analyse these screens, a previously formidable challenge, and (3) completer genome-wide experiments to identify genes involved in the TSC pathway.

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