University of Minnesota
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Researchers develop new more stable, efficient drug-delivery vehicles

Polymeric delivery vehicles offer promising biological activity for nucleic acid and genome editing therapies, which are of high interest to the field of personalized medicine. Researchers in the Department of Chemistry and Center for Genome Engineering at the University of Minnesota have published a study that demonstrates the synthesis and characterization of a new class of trehalose-based block copolycations designed to stabilize gene therapy formulations for lyophilization/storage and in vivo administration.

This research effort was led by Zachary Tolstyka, a former post-doctoral associate, and Haley Phillips, a current graduate student in the research group of Professor Theresa M. Reineke. The article entitled, “Trehalose-Based Block Copolycations Promote Polyplex Stabilization for Lyophilization and in Vivo pDNA Delivery,” is published in ACS Biomaterials Science and Engineering, and was selected as an American Chemical Society Editor’s Choice Article, allowing open access of their work to the global community of researchers.

The trehalose block copolymers developed in this study promote improved colloidal “stealth” stabilization of nanomedicines as compared to polyethylene glycol (PEG)-coated nanosystems. Nanoparticles formed from the trehalose-based polymers and plasmid DNA were found to efficiently deliver plasmid DNA via systemic injection to the mouse liver and express transgenes with high efficiency without toxic effects. These cationic glycopolymer delivery vehicles have potential for improving gene therapies and genome editing tools in preclinical animal models.