Alex's Lemonade Stand Foundation for Childhood Cancer

The goal of this project is to test a novel strategy for disrupting the effects of MYCN amplification, which is seen in over 50% of high risk neuroblastoma patients. Although the association of MYCN amplification with a poor outcome is well established, attempts to directly target MYCN have been challenging. Therefore, we propose to tackle one of the consequences of amplified MYCN that enables abnormal cell growth, that of increased protein expression (translation). Proteins are the “doers” in the cell and originate from RNA.

Mentor Name: Steve DuBois

Mentor Name: Pratiti Bandopadhayay

During my summer internship, I will study how the different effects each histone mutation has on DNA repair. To do this, we will use model systems that the lab has developed to introduce histone mutations into normal cells. I will use a range of molecular biology methods to study the effect of these mutations on normal cells and the subsequent changes in normal cellular processes, including different DNA repair responses.

Project Team

Runt-related transcription factor 1 (RUNX1) plays key regulatory roles in blood cell development and disease. Inherited mutations in RUNX1 gene results in familial platelet disorder with predisposition to AML (FPD/AML), a condition characterized by easy bleeding or bruising as a result of low platelet levels and an increased risk of acute myeloid leukemia or other hematologic cancers in middle age.