Article

Discovery of novel compounds and target mechanisms using a high throughput, multiparametric phenotypic screen in a human neuronal model of Tuberous Sclerosis

2024-02-25

Abstract excerpt

<h4>SUMMARY</h4> Tuberous sclerosis complex (TSC) is a rare genetic disorder caused by mutations in the mTOR pathway genes TSC1 or TSC2 . TSC can affect multiple organs including the brain, and most patients (75-90%) present with seizures during early childhood and intractable epilepsy throughout life. mTOR inhibitors, part of the current standard of care, lack the optimal characteristics to fully address patie...

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Literature Corpus work: da3b6d85-5ed0-5cdd-8749-f5e47fec7f9f
DOI: 10.1101/2024.02.22.581652

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