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Nucleic Acid-Based Nanoconstructs for the Treatment of Cancer
Northwestern University

Center Overview

Current cancer treatment methodologies fall short of providing efficacious, targeted, precision therapies geared towards the individual patient. Due to their novel chemical, biological, and physical properties, nucleic-acid based nanoconstructs can be used to gain access to privileged intracellular environments, discover new aspects of cancer biology, and exploit nanostructure-biomolecular interactions to create effective treatment options. The Northwestern CCNE will explore these vast possibilities by applying a novel class of nanostructure genetic constructs - the spherical nucleic acid (SNA) and variants of it - for the study and treatment of brain and prostate cancer.

SNAs enter cells in large quantities as single-entity agents and have been shown to cross the blood-brain-barrier (BBB) and the blood-tumor-barrier (BTB) in a systemic delivery scheme, and the epidermis in a topical approach. Once inside the cellular environment, SNAs resist enzymatic degradation, do not elicit toxicity or immunogenicity, and can be used to regulate gene expression via antisense and RNAi pathways. SNAs form the foundation of the technological focus of the Northwestern CCNE which, once successful, will generate a large body of fundamental knowledge that will stand to unravel a portion of the complex landscape of cancer genetics and biology, and deliver two first-in-class SNA nanostructure-based agents - a therapeutic for precision neuro-oncology that can be used to treat glioblastoma multiforme and a therapeutic cancer vaccine that can be used to treat prostate cancer.

The Northwestern CCNE is led by International Institute for Nanotechnology Director, Chad A. Mirkin, PhD, and Robert H. Lurie Comprehensive Cancer Center Director, Leonidas Platanias, MD. Dr. Mirkin and Dr. Platanias bring extensive knowledge, skill, and experience to the enterprise, and together, share the authority and responsibility for leading and directing the project.