Gene therapy is an approach for human disease treatment. This is done by inserting nucleic acids into cells to produce proteins, fix or replace damaged genes, and inhibit expression of harmful genes. The gene delivery systems are classified into viral and nonviral systems. There are several advantages in nonviral delivery systems, including biological safety (safer than viral vehicle), low immunogenicity, ability to deliver large genes, and large scale production at a reasonable cost. However, non-viral delivery systems have barriers that need to be overcome such as: biocompatibility of polymers used (toxicity) transfection efficiency, nuclear localization, and packaging and unpackaging of DNA. This invention addresses some of these issues.
This technology uses a multi-component approach to gene delivery that allows DNA binding strength, endosomolytic capabilities and targeting to be tuned separately in the resulting vector.
- Pharmaceutical industry
- Gene delivery/therapy companies
- Companies selling research reagents
- Better transfection efficiency compared to other gene therapy delivery systems
- Easier: Does not require chemical reactions to assemble the supramolecule
- Able to arrive at a useful delivery vehicle using combination at a faster rate.