Boston Children’s Hospital

Familial RUNX1 disorders [familial platelet disorder (FPD)/ acute myeloid leukemia (AML)] are caused by inherited mutations in one of the two copies of the RUNX1 gene. The RUNX1 gene produces a protein that normally turns on and off other key genes required for normal blood cell development. Individuals with FPD/AML have low platelet counts and increased leukemia risk. Because the leukemia onset takes time, there is a window of opportunity to intervene to prevent leukemia development.

Modeling RUNX1-Associated Clonal Hematopoietic Disorders in Zebrafish

Acute Myeloid Leukemia (AML)

Background

The balanced function of blood stem cells ensures normal production of blood cells. Individuals with inherited mutations in the RUNX1 gene have an increased lifetime risk of developing blood malignancies rising from the imbalance of these cells due to an abnormal clone. The clonal disorders result in ineffective production of blood cells and are on a spectrum from pre-leukemia, such as myelodysplastic syndromes (MDS), to acute myeloid leukemia (AML). For clonal disorders to initiate, genetic abnormalities in addition to RUNX1 are required.

GeneEditing in T cell Leukemia to Promote Immunomodulatory Forms of Cell Death

Leukemia, Lymphoma

Background

Transcriptional Effectors of Activated RAS Signaling in Juvenile Myelomonocytic Leukemia

Juvenile Myelomonocytic Leukemia (JMML)

Background

Juvenile myelomonocytic leukemia (JMML) is an aggressive blood cancer of young children, typically affecting those less than 4 years of age. The only current curative treatment is bone marrow transplantation. Yet, despite this very aggressive therapy about 50% of children still ultimately die from their disease. Thus, more effective and less toxic treatments are urgently needed.

Regulation of Hematopoietic Stem Cell Engraftment by the Endothelial Cell Niche

Leukemia

Background

Allogeneic hematopoietic stem cell transplantation (HSCT), or bone marrow transplantation, is the most aggressive treatment available for children with high-risk or relapsed leukemia. Despite this aggressive treatment, half of the children who receive HSCT will ultimately die of their disease or the side effects of transplantation. Consequently it is important for us to identify new ways to improve the safety and efficacy of HSCT.

A Description of Symptom Experience and Biological Covariates in Children with Acute Lymphocytic Leukemia

Acute Lymphoblastic Leukemia (ALL)

Alex's Lemonade Stand Foundation for Childhood Cancer