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Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo.

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.

SUBMITTER: Dong Y

PROVIDER: S-EPMC5278877 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature

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Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo.

Dong Yizhou Y Dorkin J Robert JR Wang Weiheng W Chang Philip H PH Webber Matthew J MJ Tang Benjamin C BC Yang Junghoon J Abutbul-Ionita Inbal I Danino Dganit D DeRosa Frank F Heartlein Michael M Langer Robert R Anderson Daniel G DG

Nano letters 20160113 2

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 e ...[more]

PMID: 26727632

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