Integration of Whole-Genome Sequencing Analysis with Unique Patient-Derived Models Reveals Clinically Relevant Drug Targets in TFCP2 Fusion-Defined Rhabdomyosarcoma.

ABSTRACT: Precision medicine is a likely future for all cancer treatment but may have its greatest impact on less common, high-mortality, and molecularly heterogeneous cancers. TFCP2-rearranged rhabdomyosarcoma (RMS) is a rare, aggressive cancer with poor survival due to the lack of effective therapies and relevant models to facilitate research. In this study, we establish the first matched patient-derived xenograft and cell line model for TFCP2-rearranged intraosseous RMS, coupled with comprehensive multiomic and functional analyses, to discover and preclinically validate novel actionable molecular targets for this malignancy. Sequencing analyses of matched patient tumor and xenograft material revealed alterations in gene networks associated with the oncogenic, potentially targetable PI3K/AKT pathw

SUBMITTER: Bergsma P 

PROVIDER: S-EPMC12670081 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature