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University of Pennsylvania

3451 Walnut Street
Philadelphia, PA 19004
United States

B cell acute lymphoblastic leukemia (B-ALL) is the most common cancer in children. Despite significant improvements in chemotherapy combinations, approximately 20% of children with high-risk B-ALL will fail their treatments. It is now clear that there are different subtypes of ALL with different genetic mutations. These mutations activate and disrupt pathways within the leukemia cells that allows the cells to grow out of control.

Background

Background

Acute myeloid leukemia (AML) is the most life-threatening form of leukemia requiring the most intensive treatment. While advances in its treatment have improved survival, chemotherapy exposure results in significant side effects. One type of drug, anthracyclines, increases the risk for heart complications.

Background

In 30-percent of neuroblastoma cases aberrant activity of the MYCN-gene is present. Current therapies for high-risk MYCN-driven neuroblastoma are ineffective, and only 40% of children with disease can achieve long-term event-free survival. These realities underscore the urgent need to discover novel therapeutic strategies that are more effective in combating neuroblastoma.

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