title: mRNA transcript therapy.
abstract: mRNA is the central molecule of all forms of life. It is generally accepted that current life on Earth descended from an RNA world. mRNA, after its first therapeutic description in 1992, has recently come into increased focus as a method to deliver genetic information. The recent solution to the two main difficulties in using mRNA as a therapeutic, immune stimulation and potency, has provided the basis for a wide range of applications. While mRNA-based cancer immunotherapies have been in clinical trials for a few years, novel approaches; including, in vivo delivery of mRNA to replace or supplement proteins, mRNA-based generation of pluripotent stem cells, or genome engineering using mRNA-encoded meganucleases are beginning to be realized. This review presents the current state of mRNA drug technologies and potential applications, as well as discussing the challenges and prospects in mRNA development and drug discovery.
authors.affiliation: Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
authors.forename: Drew
authors.initials: D
authors.lastname: Weissman
mesh_terms: D016503:Drug Delivery Systems; D015195:Drug Design; D055808:Drug Discovery; D015316:Genetic Therapy; D006801:Humans; D007167:Immunotherapy; D039904:Pluripotent Stem Cells / Q000378:metabolism; D011506:Proteins / Q000378:metabolism; D011560:Pseudouridine; D020871:RNA Stability; D012333:RNA, Messenger / Q000008:administration & dosage / Q000235:genetics / Q000276:immunology*; D013678:Technology, Pharmaceutical; D019444:Vaccines, DNA / Q000008:administration & dosage / Q000235:genetics / Q000276:immunology*
chemical_list: D011506:Proteins; D012333:RNA, Messenger; D019444:Vaccines, DNA; D011560:Pseudouridine
keywords: PKR; mRNA; modified nucleoside; pseudouridine; purification; therapeutic protein; vaccine