Pharmaceutical Sciences Seminar Series

Nanoparticles for Safe and Efficient Delivery of Nucleic Acids and CRISPR Genome Editors

(Drug DELIVERY Specific Seminar)

• Sarah Gong, PhD
• University of Wisconsin-Madison

Gene therapy, particularly, genome editing therapy can potentially be used to treat many genetic diseases including some currently without a cure. However, one major hurdle to the clinical translation of gene therapy is the lack of safe and efficient delivery approaches for nucleic acids and genome editors. The Gong lab recently developed several types of patent-pending nanoplatforms, which can be used to deliver a wide range of biologics including nucleic acids and genome editors both in vitro and in vivo. These nanoplatforms possess a number of desirable characteristics including good biocompatibility, high loading content and loading efficiency, excellent in vivo stability before reaching the target cell, high specificity to target tissue/cell, high cellular uptake, efficient endosomal escape capability, and rapid release of the cargo inside the cytosol via a stimuli-responsive release mechanism. The delivery efficiency and biosafety of these nanoplatforms targeting various organs including the brain, eyes, liver, lungs and muscles in mouse models have been examined.

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