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

Yizhou Dong; J. Robert Dorkin; Weiheng Wang; Philip H. Chang; Matthew J. Webber; Benjamin C. Tang; Junghoon Yang; Inbal Abutbul-Ionita; Dganit Danino; Frank Derosa; Michael Heartlein; Robert Langer; Daniel G. Anderson

doi:10.1021/acs.nanolett.5b02428

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

Yizhou Dong, J. Robert Dorkin, Weiheng Wang, Philip H. Chang, Matthew J. Webber, Benjamin C. Tang, Junghoon Yang, Inbal Abutbul-Ionita, Dganit Danino, Frank Derosa, Michael Heartlein, Robert Langer, Daniel G. Anderson^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

95 Scopus citations

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.

Original language	English
Pages (from-to)	842-848
Number of pages	7
Journal	Nano Letters
Volume	16
Issue number	2
DOIs	https://doi.org/10.1021/acs.nanolett.5b02428
State	Published - 10 Feb 2016
Externally published	Yes

Keywords

erythropoietin
mRNA
nanomaterial
polymer brush
siRNA

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10.1021/acs.nanolett.5b02428

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title = "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.",

keywords = "erythropoietin, mRNA, nanomaterial, polymer brush, siRNA",

author = "Yizhou Dong and Dorkin, {J. Robert} and Weiheng Wang and Chang, {Philip H.} and Webber, {Matthew J.} and Tang, {Benjamin C.} and Junghoon Yang and Inbal Abutbul-Ionita and Dganit Danino and Frank Derosa and Michael Heartlein and Robert Langer and Anderson, {Daniel G.}",

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T1 - Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo

AU - Dong, Yizhou

AU - Dorkin, J. Robert

AU - Wang, Weiheng

AU - Chang, Philip H.

AU - Webber, Matthew J.

AU - Tang, Benjamin C.

AU - Yang, Junghoon

AU - Abutbul-Ionita, Inbal

AU - Danino, Dganit

AU - Derosa, Frank

AU - Heartlein, Michael

AU - Langer, Robert

AU - Anderson, Daniel G.

PY - 2016/2/10

Y1 - 2016/2/10

N2 - 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.

AB - 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.

KW - erythropoietin

KW - mRNA

KW - nanomaterial

KW - polymer brush

KW - siRNA

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SN - 1530-6984

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JO - Nano Letters

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

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Keywords

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