Unmet NeedGene therapy has the potential to offer a one-time cure for severe, debilitating diseases that were previously untreatable or required chronic therapy. While a few viral-based gene therapies have recently received FDA approval, safety concerns around viral-based gene therapies continue to limit their clinical development. Non-viral strategies of gene delivery, particularly nanoparticle-based approaches, may provide several advantages relative to viral delivery, including enhanced safety profiles, cost-effective production, and more localized gene expression. However, the clinical applications of non-viral gene carriers are limited by low
in vivo transfection efficiency, which limits therapeutic efficacy. Consequently, to fully harness the potential of nanoparticle gene delivery platforms, control over their circulation, tissue distribution, and cellular uptake must be controlled and improved.
Technology OverviewJHU researchers have developed a method to generate improved non-viral, nucleic acid-containing nanoparticles for gene delivery applications. The inventors identified particle assembly conditions and a graft copolymer structure that can be used to control the shape of these polymeric micellar nanoparticles. Importantly, these nucleic acid-encapsulating nanoparticles show a high level of transfection efficiency when injected through intravenous injection, without detectable toxicity. The inventors demonstrated that these particles were able to efficiently transfect DNA or RNA both
in vivo and
in vitro. A cell targeting ligand can also be conjugated to these nanoparticles, enabling the specific targeting of these nanoparticles to a certain cell type.
Stage of DevelopmentThis nanoparticle platform will enable the development of improved nonviral nanoparticles for
in vitro and
in vivo gene medicine delivery.
PublicationsWilliford JM, et al. Critical Length of PEG Grafts on lPEI/DNA Nanoparticles for Efficient in Vivo Delivery. ACS Biomater Sci Eng. 2016 Apr 11; 2(4):567-578.
