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Targeted Inhibition of Lin28/let-7 Binding to Treat Pediatric Malignancy

Boston Children’s Hospital
George Q. Daley, MD/PhD
Grant Type: 
Innovation Grants
Year Awarded: 
Type of Childhood Cancer: 
Hepatoblastoma, Neuroblastoma, Wilms Tumor
Project Description: 


The LIN28 family of RNA binding proteins are known drivers of many pediatric cancers. These proteins function by blocking the function of the let-7 family of microRNAs, which exert tumor suppressive effects. To date, despite the panoply of childhood tumors driven by LIN28, efforts to target this protein in pediatric cancer are lacking.

Project Goal

We propose a series of studies aimed to develop novel drugs targeting these proteins. Our collaborator has identified a series of candidate LIN28 inhibitory compounds in biochemical assays that we will investigate as LIN28 inhibitors in cells and animal models of pediatric tumors.

Additionally, we will search for known drugs that have characterized patterns of absorption and distribution in mammals that have inhibitory activity against LIN28 in tumor models. Together, these translational studies will begin to address the viability of inhibition of LIN28 as a therapeutic approach in childhood malignancy. We believe that these studies are within the mission of Alex's Lemonade Stand Foundation as they aim to accelerate translation of findings in disease models into viable therapeutics.