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

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.

• Publication year

  2019

• Venue

  Molecular Therapy

• Publication date

  2019-04-01

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1016/j.ymthe.2019.02.019PMID 30905577PMCID PMC6453549
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium

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  2018influential reference
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