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

被引：97

作者：

Dong, Yizhou ^{[1
,2
,5
,9
]}

Dorkin, J. Robert ^{[3
]}

Wang, Weiheng ^{[1
,5
]}

Chang, Philip H. ^{[1
,5
]}

Webber, Matthew J. ^{[1
,5
]}

Tang, Benjamin C. ^{[1
,5
,10
]}

Yang, Junghoon ^{[1
]}

Abutbul-Ionita, Inbal ^{[6
,7
]}

Danino, Dganit ^{[6
,7
]}

DeRosa, Frank ^{[8
]}

Heartlein, Michael ^{[8
]}

Langer, Robert ^{[1
,2
,4
,5
]}

Anderson, Daniel G. ^{[1
,2
,4
,5
]}

机构：

[1] MIT, David H Koch Inst Integrat Canc Res, 77 Massachusetts Ave, Cambridge, MA 02139 USA

[2] MIT, Dept Chem Engn, Cambridge, MA 02139 USA

[3] MIT, Dept Biol, 77 Massachusetts Ave, Cambridge, MA 02139 USA

[4] MIT, Inst Med Engn & Sci, 77 Massachusetts Ave, Cambridge, MA 02139 USA

[5] Harvard Univ, Sch Med, Childrens Hosp Boston, Dept Anesthesiol, Boston, MA 02115 USA

[6] Technion Israel Inst Technol, Dept Biotechnol & Food Engn, IL-32000 Haifa, Israel

[7] Russell Berrie Nanotechnol Inst, IL-32000 Haifa, Israel

[8] Shire, 300 Shire Way, Lexington, MA 02421 USA

[9] Ohio State Univ, Coll Pharm, Div Pharmaceut & Pharmaceut Chem, 500 W 12th Ave, Columbus, OH 43210 USA

[10] Amgen Inc, Dept Pharmacokinet & Drug Metab, 1120 Vet Blvd, San Francisco, CA 94080 USA

来源：

NANO LETTERS | 2016年 / 16卷 / 02期

基金：

美国国家卫生研究院;

关键词：

siRNA; mRNA; polymer brush; erythropoietin; nanomaterial; LIPID NANOPARTICLES; GENE DELIVERY; PSEUDOURIDINE; THERAPEUTICS; RECOGNITION; EXPRESSION; EFFICIENCY; PROGRESS; POTENT;

D O I：

10.1021/acs.nanolett.5b02428

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

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.

引用

页码：842 / 848

页数：7

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