Gene therapy holds the promise of providing single treatment curative benefits for diseases that currently require repeated infusions. The majority of clinical gene therapies make use of viral vectors which are limited by potential mutagenesis, immunogenicity, and non-specific delivery. Non-viral gene therapy utilizing microbubbles (MBs) and ultrasound has the potential to address these limitations.<br/>MBs are clinically used ultrasound contrast agents that have been frequently cited as promising vehicles for targeted drug delivery. It has been shown that a collapsing bubble will produce local shock waves, water jets, and shear forces that will permeabilize nearby cell membranes, allowing the direct delivery of nucleic acids or other therapeutics into the cell’s cytosol. This phenomenon, termed sonoporation, will not only allow for spatial control of delivery using ultrasound but will also allow for high delivery efficiency since the drug will be bypassing endocytosis. Several examples of gene delivery utilizing cationic MBs have already been reported in the literature. However, due to their limited surface area and poor interaction, these cationic MBs only weakly bind DNA. Conjugating biocompatible cationic polymers onto the surface of MBs will not only increase DNA binding due to a larger surface area, but will also shield the DNA from serum proteins.<br/>Here we report the development of polyplex-conjugated microbubbles utilizing spermine-conjugated dextran as a non-toxic polymer to complex DNA. Conjugating cationic polymers onto the surface of MBs will allow for tighter binding and increased loading of DNA as it will intertwine with the polymer instead of lying flat on the surface of the bubble. Using this platform, we achieved a 10-fold increase in DNA packing as compared to previous cationic MB formulations and are currently exploring its application to other nucleic acids.<br/>

Development of novel polyplex-conjugated microbubbles as vectors for gene delivery

Macromolecular Chemistry: The Second Century

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ACS Spring 2020 National Meeting & Expo

The official archive and repository for all ACS Meetings & Expositions abstracts and select posters or presentations.