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RNA Innovation Seminar: Alexandra Piotrowski-Daspit, Internal Medicine
Monday, June 3 @ 4:00 pm - 5:00 pm
“RNA: the delivery perspective”
Alexandra Piotrowski-Daspit, Ph.D.
Assistant Professor, Biomedical Engineering
Assistant Professor, Internal Medicine – Pulmonary & Critical Care Medicine Division
University of Michigan
In-person: BSRB, Kahn Auditorium / hybrid link
Abstract: The primary barrier for clinical translation of RNA therapeutics remains delivery to target tissues in vivo. Upon entering the body, delivery vehicles encounter extracellular and intracellular barriers. It is also unclear how carrier design features impact physiological interactions with biological systems, highlighting the need for studies that elucidate these structure-function relationships. In this seminar, I will describe our work on developing polymeric nanoparticles (NPs) to deliver therapeutic RNAs to a variety of tissues following local and systemic IV administration. We find that gene therapy can be significantly enhanced using a new class of polymeric vehicles consisting of poly(amine-co-ester) (PACE) polymers that are designed for safe and effective nucleic acid delivery. We have also developed tools to study nanomaterial-biology interactions in animal models, including a high-throughput quantitative microscopy-based platform to measure circulation half-life and biodistribution in vivo. This tool, alongside the development of novel polymeric carriers, can be used to study the structure-function relationships that guide the physiological fate of NPs in order to rationally design more effective delivery vehicles for RNA delivery.
Keywords: RNA therapeutics, in vivo delivery, polymeric nanoparticles (NPs), nanomaterial-biology interactions