Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

被引：788

作者：

Kowalski, Piotr S. ^{[1
,2
]}

Rudra, Arnab ^{[1
,2
,3
]}

Miao, Lei ^{[1
,2
]}

Anderson, Daniel G. ^{[1
,2
,3
,4
,5
]}

机构：

[1] MIT, David H Koch Inst Integrat Canc Res, 500 Main St, Cambridge, MA 02142 USA

[2] MIT, Dept Chem Engn, Cambridge, MA 02142 USA

[3] Boston Childrens Hosp, Dept Anesthesiol, 300 Longwood Ave, Boston, MA 02115 USA

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

[5] MIT, Harvard & MIT Div Hlth Sci & Technol, 77 Massachusetts Ave, Cambridge, MA 02139 USA

来源：

MOLECULAR THERAPY | 2019年 / 27卷 / 04期

关键词：

COLONY-STIMULATING FACTOR; T-CELL RESPONSES; IN-VIVO; LIPID NANOPARTICLES; INTRACELLULAR DELIVERY; SIRNA DELIVERY; MEDIATED DELIVERY; CATIONIC LIPIDS; GENE DELIVERY; NONVIRAL DELIVERY;

D O I：

10.1016/j.ymthe.2019.02.012

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems. In this review, we discuss the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and we present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination.

引用

页码：710 / 728

页数：19

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