Dana-Farber Cancer Institute

Dana-Farber Cancer Institute

44 Binney Street

Boston, MA 2115

United States

Divergent Core Transcriptional Regulatory Circuitries to Highlight Context-Specific Vulnerabilities in AML

Background

Acute myeloid leukemia (AML) is one of the deadliest pediatric cancers, responsible for 10% of total cancer mortality in children. AML is caused by mutations in normal bone marrow stem cells that make them grow out of control (i.e. become malignant). There are many AML-causing mutations and they result in many different subtypes of AML with very different responsiveness to treatment and outcomes. We need a better mechanistic understanding of both common and divergent (i.e. subtype-specific) pathways leading to AML.

MYC-driven Core Regulatory Circuits in Neuroblastoma

Neuroblastoma

Novel Approaches to AML Differentiation Therapy

Acute Myeloid Leukemia (AML)

Survival of children with AML remains poor because our treatments haven't changed much in 30 years. AML is a disease of DNA – mutations found in leukemia DNA are not seen in normal blood cells. However, we think that the physical structure of DNA itself could also be important in leukemia development. If the DNA in a cell was stretched out, it would be six feet long, yet it has to be tightly packed to fit inside the head of a pin. When we looked at leukemia under the microscope, the DNA was not as tightly wound as it should be.

Reversing mSWI/SNF (BAF) Complex Misassembly and Genomic Targeting in Human Synovial Sarcoma

Synovial Sarcoma

Targeting BRD9 in Sarcoma

Synovial Sarcoma

Characterizing Novel Rearrangements in Diffuse Intrinsic Pontine Glioma

Diffuse Intrinsic Pontine Glioma (DIPG)

Background

Diffuse intrinsic pontine gliomas (DIPG) are devastating childhood brain tumors. There are no known curative treatments for DIPG. DIPGs arise in a delicate part of the brain known as the brainstem, rendering them un-resectable. Even biopsy of the tumors has been historically avoided due to the critical function of the brainstem. The lack of biopsy samples has greatly hampered understanding of the biology of the tumors.