Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung Diseases

被引：69

作者：

Sahu, Itishri ^{[1
]}

Haque, A. K. M. Ashiqul ^{[1
]}

Weidensee, Brian ^{[1
]}

Weinmann, Petra ^{[1
]}

Kormann, Michael S. D. ^{[1
]}

机构：

[1] Univ Tubingen, Univ Childrens Hosp, Dept Pediat Pediat Infectiol & Immunol 1, Translat Genom & Gene Therapy, Wilhelmstr 27, D-72074 Tubingen, Germany

来源：

MOLECULAR THERAPY | 2019年 / 27卷 / 04期

基金：

欧洲研究理事会;

关键词：

PRIMARY CILIARY DYSKINESIA; REGULATORY T-CELLS; PULMONARY NEUROENDOCRINE CELLS; SURFACTANT REPLACEMENT THERAPY; BRONCHIOALVEOLAR STEM-CELLS; CIGARETTE-SMOKE EXPOSURE; CYSTIC-FIBROSIS SPUTUM; MEDIATED GENE-TRANSFER; SP-B; MOUSE MODEL;

D O I：

10.1016/j.ymthe.2019.02.019

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：

Protein supplementation therapy using in vitro-transcribed (IVT) mRNA for genetic diseases contains huge potential as a new class of therapy. From the early ages of synthetic mRNA discovery, a great number of studies showed the versatile use of IVT mRNA as a novel approach to supplement faulty or absent protein and also as a vaccine. Many modifications have been made to produce high expressions of mRNA causing less immunogenicity and more stability. Recent advancements in the in vivo lung delivery of mRNA complexed with various carriers encouraged the whole mRNA community to tackle various genetic lung diseases. This review gives a comprehensive overview of cells associated with various lung diseases and recent advancements in mRNA-based protein replacement therapy. This review also covers a brief summary of developments in mRNA modifications and nanocarriers toward clinical translation.

引用

页码：803 / 823

页数：21

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