Abstract
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.
| Original language | English |
|---|---|
| Pages (from-to) | 710-728 |
| Number of pages | 19 |
| Journal | Molecular Therapy |
| Volume | 27 |
| Issue number | 4 |
| DOIs | |
| Publication status | Published - 10 Apr 2019 |
| Externally published | Yes |
UN SDGs
This output contributes to the following UN Sustainable Development Goals (SDGs)
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SDG 3 Good Health and Well-being -
SDG 9 Industry, Innovation, and Infrastructure
Keywords
- biomaterials
- clinical trial
- CRISPR
- gene editing
- gene therapy
- lipid nanoparticles
- mRNA delivery
- mRNA nanoparticles
- mRNA vaccine
- protein replacement
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