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Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo
Yizhou Dong1,2, J Robert Dorkin, Weiheng Wang1,2
1David H. Koch Institute for Integrative Cancer Research, ‡Department of Chemical Engineering, §Department of Biology, and ∥Institute for Medical Engineering and Science, Massachusetts Institute of Technology , Cambridge, Massachusetts 02139, United States.
Abstract:
Safe and effective delivery is required for siRNA and mRNA-based therapeutics to reach their potential. Here, we report on the development of poly(glycoamidoamine) brush nanoparticles as delivery vehicles for siRNA and mRNA. These polymers were capable of significant delivery of siRNA against FVII and mRNA-encoding erythropoietin (EPO) in mice. Importantly, these nanoparticles were well-tolerated at their effective dose based on analysis of tissue histology, systemic cytokine levels, and liver enzyme chemistry. The polymer brush nanoparticles reported here are promising for therapeutic applications.
