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RESEARCH PRODUCT

mRNA-based therapeutics — developing a new class of drugs

ÖZlem TüreciUgur SahinKatalin Karikó

subject

Pluripotent Stem CellsPharmacologyDrugImmunogenicitymedia_common.quotation_subjectProteinsGeneral MedicineComputational biologyBiologyPharmacologyGenome engineeringDrug Delivery SystemsDrug classProtein replacement therapyDrug developmentInfectious disease (medical specialty)Drug DesignDrug DiscoveryAnimalsHumansImmunotherapyRNA MessengerInduced pluripotent stem cellmedia_common

description

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 infectious disease vaccines have entered clinical development. Meanwhile, emerging novel approaches include in vivo delivery of IVT mRNA to replace or supplement proteins, IVT mRNA-based generation of pluripotent stem cells and genome engineering using IVT mRNA-encoded designer nucleases. This Review provides a comprehensive overview of the current state of mRNA-based drug technologies and their applications, and discusses the key challenges and opportunities in developing these into a new class of drugs.

yearjournalcountryeditionlanguage
2014-09-19Nature Reviews Drug Discovery
https://doi.org/10.1038/nrd4278