Dissertations, Theses, and Capstone Projects

Date of Degree

6-2024

Document Type

Dissertation

Degree Name

Ph.D.

Program

Biochemistry

Advisor

Stephen Redenti

Committee Members

Avi Ma’ayan

Julio Gallego-Delgado

Liang Zhao

Pratyusha Mandal

Subject Categories

Biochemistry | Molecular Biology

Keywords

Extracellular Vesicles(EV); Retinoblastoma; Cancer metastasis; QSAR-ML; Drug repurposing

Abstract

Extracellular vesicles (EVs) are lipid-enclosed particles that are generated by most types of cells and have been identified as an important mechanism for cell communication aside from cell direct contact. EVs contain a variety of functional macromolecules including proteins, lipids, RNA, which can be transferred from the host cells to target cells at a distance. The molecular pathways of EV biogenesis have been studied intensely in recent decades and the process involves several key genes and proteins. Cancer cell released EVs target distant tissues and upregulate signals that are attractive to cancer cell homing, playing a role in tumor progression and metastasis. Blocking the release of EVs from cancer cells with targeted drugs is predicted to reduce metastatic signaling and disease pathogenesis, with the potential to reduce recurrence. In this study, we first created a focused database of genes and drugs that have been used by researchers to inhibit EV release. Second, we developed a QSAR-ML model to identify novel drugs with functional properties similar to existing EV release-inhibition drugs. Third, QSAR-ML identified novel drugs were validated in vitro for inhibition of EV release from childhood ocular cancer retinoblastoma cells. This work provides a novel computational tool and a novel drug library that may optimize therapeutic strategies to reduce EV-mediated signaling and retinoblastoma pathogenesis.

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