The RUNX1 Research Program

The RUNX1 Research Program is a non-profit organization committed to finding a cure for RUNX1-FPD and to support patients in the RUNX1 community by providing a forum in which to help connect, inform and educate patients on the disorder. In addition to providing grants for relevant research projects, the program hopes to build public awareness, educating both patients and healthcare providers on the latest research regarding the disorder.

RUNX1 FPD is a hereditary blood disorder which predisposes an individual to acquiring leukemia in his or her lifetime. The disorder carries a 50 percent lifetime risk of progressing to leukemia through the acquisition of additional mutations in other genes.

RUNX1 Research Program

Co-Funded Projects

Project Title	Institutions	Co-Funder(s)
Modeling Clonal Hematopoiesis and Genetic Correction in RUNX1-FPD Using Human iPSC-derived HSCs	Icahn School of Medicine at Mount Sinai	The RUNX1 Research Program
Therapeutic targeting of clonal hematopoiesis in RUNX1 mutant FPDMM	University of Pennsylvania	The RUNX1 Research Program
Mutagenesis and disease progression in RUNX1 mutant blood stem cells is caused by inflammation-induced hyperactive signaling and supraphysiological mitochondrial ROS	St. Jude Children’s Research Hospital	The RUNX1 Research Program
Creating an Infrastructure for Collaborative Clinical Research and Publication of Consensus Guidelines for RUNX1 Familial Platelet Disorder	The Cleveland Clinic Foundation	The RUNX1 Research Program
Using Chemical Genetics to Define the Precise Role of RUNX1 in Transcription and Beyond	Albert Einstein College of Medicine	The RUNX1 Research Program
Delivering on the Promise of Germline Genetic Testing for RUNX1 Variants	University of Washington, Seattle	The RUNX1 Research Program
Role of PHF6 Mutations in Germline RUNX1 Deficiency Associated Hematopoietic Disorders	The Regents of the University of California San Francisco	The RUNX1 Research Program
Characterizing Inflammatory Phenotypes Associated with RUNX1 Deficiency	Dana-Farber Cancer Institute	The RUNX1 Research Program
Modeling familial platelet disorder associated RUNX1 mutations in mice	University of Massachusetts Medical School	The RUNX1 Research Program
Assessing Clonal Fitness and Mechanisms of Clonal Evolution in FPD-MN	Memorial Sloan-Kettering Cancer Center	The RUNX1 Research Program

Project Title

Institutions

Co-Funder(s)

Modeling Clonal Hematopoiesis and Genetic Correction in RUNX1-FPD Using Human iPSC-derived HSCs

Icahn School of Medicine at Mount Sinai

The RUNX1 Research Program

Therapeutic targeting of clonal hematopoiesis in RUNX1 mutant FPDMM

University of Pennsylvania

The RUNX1 Research Program

Mutagenesis and disease progression in RUNX1 mutant blood stem cells is caused by inflammation-induced hyperactive signaling and supraphysiological mitochondrial ROS

St. Jude Children’s Research Hospital

The RUNX1 Research Program