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This post is Part 2 of a series of Zone blogs featuring DNA plasmids used to produce in vitro transcribed (IVT) mRNA. Part 1 , posted on June 8, 2021, provided historical perspectives on the discovery of plasmids, as well as descriptions of how these circular double-stranded DNAs enabled recombinant DNA technology and now IVT mRNA production. The two growth phases of plasmid applications are reflected in this chart of publications in PubMed indexed to gene therapy and genetic vacci...
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TL mRNA Therapeutic for Treatment of Argininosuccinic Aciduria, an Inherited Rare Metabolic Disorder
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Introduction Synthetic mRNA produced by in vitro transcription (IVT) is the active pharmaceutical ingredient of approved vaccines and of many drugs under development. IVT mRNA typically contains several hundred bases of non-coding untranslated regions (UTRs) that are involved in the stabilization and translation of the mRNA. Remarkably, new research has discovered that these UTRs can be virtually eliminated without compromising translation of the encoded protein of interest. ...
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