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

Abstract

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.

Brief Biography

Dr. Shaoqin “Sarah” Gong is a Vilas Distinguished Achievement Professor, Advancing Vision Science Chair Professor, and Retina Research Foundation Edwin and Dorothy Gamewell Professor in the Departments Ophthalmology and Visual Sciences and Biomedical Engineering at the University of Wisconsin–Madison. She received dual bachelor’s degrees from Tsinghua University in Materials Science and Engineering and Economics and Management in 1991. She earned a master’s degree from Tsinghua University in 1994 and a PhD degree from the University of Michigan–Ann Arbor in 1999 both in Materials Science and Engineering. Prof. Gong has co-authored nearly 200 peer-reviewed journal articles with an H-index of 74. She is an editorial board member for several journals including Biomaterials, Theranostics, Biofabrication, and Nanotheranostics. She is also a member of the advisory board for Biomaterials Science and served as an Associate Editor for Biomaterials. Prof. Gong has received a number of awards including the NSF CAREER Award and NIH Research Development Award. She is a Fellow of the American Institute for Medical and Biomedical Engineering. Prof. Gong’s current research focuses on the development of novel biomaterials for various biomedical applications including nanomedicines and tissue engineering scaffolds.