mRNA-based therapeutics — developing a new class of drugs

  title={mRNA-based therapeutics — developing a new class of drugs},
  author={Uğur Şahin and Katalin Karik{\'o} and {\"O}zlem T{\"u}reci},
  journal={Nature Reviews Drug Discovery},
In vitro transcribed (IVT) mRNA has recently come into focus as a potential new drug class to deliver genetic information. Such synthetic mRNA can be engineered to transiently express proteins by structurally resembling natural mRNA. Advances in addressing the inherent challenges of this drug class, particularly related to controlling the translational efficacy and immunogenicity of the IVTmRNA, provide the basis for a broad range of potential applications. mRNA-based cancer immunotherapies and… 
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Opportunities and challenges in the delivery of mRNA-based therapeutics with a focus on non-viral delivery systems are reviewed and perspectives on the future of this promising new type of medicine are highlighted.
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The challenges for clinical translation of RNA-based therapeutics, with an emphasis on recent advances in delivery technologies, are discussed, and an overview of the applications of RNAs for modulation of gene/protein expression and genome editing that are currently being investigated both in the laboratory as well as in the clinic are presented.
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