Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung Diseases
Open accessAbstract
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.
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mRNA-based protein replacement therapies are also being explored for heart, liver, lung and other organ diseases.
“mRNA-based protein replacement therapies are also being explored for heart241, liver242, lung243 and other organ diseases12.”
From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [243]
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- Authors
- Sahu, I., Haque, A. K. M. A., Weidensee, B., Weinmann, P., & Kormann, M. S. D.
- Affiliations
- University Children's Hospital Tübingen (DE)
- Corresponding author
- Kormann, Michael S.D.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2019-04
- Volume
- 27
- Issue
- 4
- Pages
- 803-823
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC6453549
- OpenAlex
- W2921986119
- Cited by
- 104
- References
- 330
- Citations by year
- 2026: 7 · 2025: 21 · 2024: 14 · 2023: 24 · 2022: 16 · 2021: 13 · 2020: 7 · 2019: 2
- FWCI
- 0.84
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- European Research Council (637752)
- HMZ Privatstiftung
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
- Keywords
- in vitro transcribed mRNA, mRNA modification, nanocarriers, mRNA vaccines, immunogenicity reduction, clinical translation
- MeSH terms
- Animals, Humans, Lipids, Lung, Lung Diseases, Polymers, RNA Processing, Post-Transcriptional, RNA, Messenger, Transcription, Genetic, Drug Delivery Systems, Mice, Nanoparticles, Enzyme Replacement Therapy
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
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