Functional screening reveals genetic dependencies and diverging cell cycle control in atypical teratoid rhabdoid tumors.

ABSTRACT:

Background

Atypical teratoid rhabdoid tumors (ATRT) are incurable high-grade pediatric brain tumors. Despite intensive research efforts, the prognosis for ATRT patients under currently established treatment protocols is poor. While novel therapeutic strategies are urgently needed, the generation of molecular-driven treatment concepts is a challenge mainly due to the absence of actionable genetic alterations.

Results

We here use a functional genomics approach to identify genetic dependencies in ATRT, validate selected hits using a functionally instructed small molecule drug library, and observe preferential activity in ATRT cells without subgroup-specific selectivity. CDK4/6 inhibitors are among the most potent drugs and display anti-tumor efficacy due to mutual exclusive dep

SUBMITTER: Merk DJ 

PROVIDER: S-EPMC11610224 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature