Childhood Cancer

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Assessing clonal fitness and mechanisms of clonal evolution in FPD-MN

Institution: 
Memorial Sloan-Kettering Cancer Center
Researcher(s): 
Wenbin Xiao, MD/PhD
Grant Type: 
RUNX1 Early Career Investigator Grants
Year Awarded: 
2020
Type of Childhood Cancer: 
Leukemia, Acute Myeloid Leukemia (AML), Predisposition
Project Description: 

Germline RUNX1 mutations are commonly observed in familial platelet disorders (FPD); FPD patients have a high rate of transformation to myeloid neoplasms (FPD-MN). The molecular pathogenesis of FPD-MN remains elusive.

Project Goal
The proposed research here will use novel mouse models to identify contexts in which oncogenic RUNX1 mutations are necessary for disease progression and maintenance. These studies will provide insights into mechanisms of clonal fitness and leukemic transformation in FPD, which can help identify FPD patients at highest risk of transformation and inform the development of novel therapeutic approaches to prevent/intercept disease progression.

Co-funded by: 
The RUNX1 Research Program