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Modeling germline mutations in pineoblastoma uncovers lysosome disruption-based therapy

14 Apr 2020

Abstract excerpt

Pineoblastoma is a rare pediatric cancer induced by germline mutations in the tumor suppressors RB1 or DICER1. Presence of leptomeningeal metastases is indicative of poor prognosis. Here we report that inactivation of Rb plus p53 via a WAP-Cre transgene, commonly used to target the mammary gland during pregnancy, induces metastatic pineoblastoma resembling the human disease with 100% penetrance. A stabilizing...

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PMID: 32286280
DOI: 10.1038/s41467-020-15585-2

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