mRNA-based therapeutics — developing a new class of drugs

mRNA-based therapeutics — developing a new class of drugs

19 September 2014 | Ugur Sahin1,2, Katalin Kariko2,3 and Ozlem Tureci1
The article provides a comprehensive overview of mRNA-based therapeutics, highlighting their potential as a new class of drugs. In vitro transcribed (IVT) mRNA has emerged as a promising platform for delivering genetic information, with advances in addressing challenges such as translational efficacy and immunogenicity. IVT mRNA-based therapies are being explored for cancer immunotherapy, infectious disease vaccines, and protein replacement therapies. The review discusses the key principles of mRNA pharmacology, including the role of the 5' cap, poly(A) tail, untranslated regions (UTRs), and codon usage. It also covers the challenges and strategies for improving mRNA stability and translation, as well as the immune-stimulatory effects of IVT mRNA. The article outlines the progress in mRNA delivery methods, including in vitro and in vivo transfection strategies, and highlights the clinical development of IVT mRNA-based therapies in cancer immunotherapy, infectious disease vaccines, and protein replacement. The potential of mRNA-based technologies in personalized medicine and allergy treatment is also discussed.The article provides a comprehensive overview of mRNA-based therapeutics, highlighting their potential as a new class of drugs. In vitro transcribed (IVT) mRNA has emerged as a promising platform for delivering genetic information, with advances in addressing challenges such as translational efficacy and immunogenicity. IVT mRNA-based therapies are being explored for cancer immunotherapy, infectious disease vaccines, and protein replacement therapies. The review discusses the key principles of mRNA pharmacology, including the role of the 5' cap, poly(A) tail, untranslated regions (UTRs), and codon usage. It also covers the challenges and strategies for improving mRNA stability and translation, as well as the immune-stimulatory effects of IVT mRNA. The article outlines the progress in mRNA delivery methods, including in vitro and in vivo transfection strategies, and highlights the clinical development of IVT mRNA-based therapies in cancer immunotherapy, infectious disease vaccines, and protein replacement. The potential of mRNA-based technologies in personalized medicine and allergy treatment is also discussed.
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