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Nano Lett. 2016 Feb 10;16(2):842-8. doi: 10.1021/acs.nanolett.5b02428. Epub 2016 Jan 13.

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

Author information

1
David 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.
2
Department of Anesthesiology, Children's Hospital Boston, Harvard Medical School , Boston, Massachusetts 02115, United States.
3
Department of Biotechnology and Food Engineering, Technion Institute of Technology and the Russell Berrie Nanotechnology Institute , Haifa 32000, Israel.
4
Shire , 300 Shire Way, Lexington, Massachusetts 02421, 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.

KEYWORDS:

erythropoietin; mRNA; nanomaterial; polymer brush; siRNA

PMID:
26727632
PMCID:
PMC5278877
DOI:
10.1021/acs.nanolett.5b02428
[Indexed for MEDLINE]
Free PMC Article

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