Date of Award

Spring 2026

Document Type

Thesis

Degree Name

Master of Science (MS) in Biology

Department

Biology

Committee Chair

Le Su

Abstract

CIC-DUX4 sarcoma (CDS) is a rare and extremely aggressive soft tissue sarcoma that primarily affects children and young adults. CDS is characterized by an oncogenic chromosomal translocation. Current treatments for CDS are similar to those in other sarcomas like surgery, chemotherapy, and radiation. Treatments, however show that CDS responds poorly compared to other sarcomas, due to relapses of localized disease at high rates and short duration response of treatment, proving the importance of understanding CIC-DUX4’s complex role on how it regulates overall gene expression in CDS cells from a molecular approach.

UBE3A is a ubiquitin protein ligase that plays a role in protein stability through the proteasomal degradation system. Studies have shown that UBE3A is a transcriptional coactivator. It is known that UBE3A targets cellular and viral proteins that are responsible for neurodevelopmental disorders, and for the progression of viral diseases. These studies indicate that understanding UBE3A function is imperative to regulating various human and viral diseases.

In CDS cell lines, the molecular and cellular characteristics of the role UBE3A were assessed on cell proliferation, migration, and 3D tumor spheroid growth, showing that UBE3A plays an essential function in CDS and a significant role in over-expression of ETV4 and CCNE1, indicating therapeutic potential of CIC-DUX4 target genes associated with UBE3A.

Keywords: CIC-DUX4 Sarcoma, CDS Biology, UBE3A, ETV4, CCNE1

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