Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases
Open accessAbstract
Promising improvements in the field of transcript therapeutics have clearly enhanced the potential of mRNA as a new pillar for protein replacement therapies. Synthetic mRNAs are engineered to replace mutated mRNAs and to be immunologically inconspicuous and highly stable while maximizing protein expression. Approaches to deliver mRNA into the cellular cytoplasm safely and efficiently have been further developed so that two mRNA-based approaches replacing vascular endothelial growth factor (VEGF) and cystic fibrosis transmembrane conductance regulator (CFTR) have now made it into clinical trials. These studies bring mRNA therapeutics for protein replacement therapy closer to clinical realization. Herein, we provide an overview of preclinical and clinical developments of mRNA therapeutics for liver diseases.
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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 [242]
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References 109 · Cited by 115
- Authors
- Trepotec, Z., Lichtenegger, E., Plank, C., Aneja, M. K., & Rudolph, C.
- Affiliations
- Ethris (Germany) (DE)
- TUM Klinikum (DE)
- Corresponding author
- Aneja, Manish K.
- Rudolph, Carsten
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2019-04
- Volume
- 27
- Issue
- 4
- Pages
- 794-802
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC6453508
- OpenAlex
- W2906077494
- Cited by
- 115
- References
- 109
- Citations by year
- 2026: 9 · 2025: 14 · 2024: 21 · 2023: 19 · 2022: 14 · 2021: 28 · 2020: 4 · 2019: 6
- FWCI
- 0.93
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- mediaTUM – the media and publications repository of the Technical University Munich (Technical University Munich) (submittedVersion)
- Funders
- Excellence Cluster “Nanosystems Initiative Munich”
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Research and Splicing
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
- Keywords
- mRNA therapeutics, mRNA delivery, liver disease, protein replacement therapy, synthetic mRNA, clinical trials, preclinical drug development
- MeSH terms
- Animals, DNA, Humans, Lipids, Liver Diseases, Polymers, RNA, Messenger, Drug Delivery Systems, Mice, Nanoparticles, Enzyme Replacement Therapy
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