Targeting gene fusions as a therapeutic approach in pediatric cancers

Document Type

Article

Publication Date

Summer 2023

Keywords

JGM

JAX Location

In: Student Reports, Summer 2023, The Jackson Laboratory

Abstract

Gene fusions are a recurrent and disease-specific class of somatic alterations common in pediatric cancers, making them an important biomarker for both diagnosis and treatment. Previous work has identified gene fusions involved in a wide variety of pediatric cancer types and begun to develop fusion RNA-targeting therapies. The aim of this project was to optimize the development of fusion-targeting therapeutics by 1) developing an application to easily identify and prioritize fusions to target for therapeutic development, and 2) creating an EWSR1-FLI1 fusion-expressing plasmid to test novel RNA sensors. The developed application highlights recurrent fusions for future RNA sensor design, and an efficient RNA sensor design was identified through testing using EWSR1-FLI1 fusion-expressing plasmid.

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