NIH Reporter #5U01CA296350-02

Study of tyrosine kinase inhibitors as preventive therapy in RUNX1-FPD

Research project funded by the National Cancer Institute to study tyrosine kinase inhibitors as preventive therapy for familial platelet disorder related to RUNX1 mutations, including testing imatinib efficacy in preclinical models and human cells.

Description

The project involves research on familial platelet disorder (FPD) related to RUNX1 mutations and potential preventive therapy using tyrosine kinase inhibitors. It aims to understand the mechanisms of disease and test therapeutic interventions in mice and human cells, including evaluating the efficacy of imatinib in restoring DNA damage response and preventing hematologic malignancies. The project is part of a phase-II clinical trial initiative funded by the National Cancer Institute (NCI), part of the NIH.

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