Titlebook: Messenger RNA Therapeutics; Stefan Jurga,Jan Barciszewski Book 2022 The Editor(s) (if applicable) and The Author(s), under exclusive licen

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Hospital-Based RNA Therapeutics, to support IND applications and first-in-man clinical trials, as well as personalized mRNA therapeutics delivered at the point-of-care, is feasible at a modest scale of cGMP manufacturing. Advances in mRNA manufacturing science and innovations in mRNA biology are increasing the scope of mRNA clinical applications.

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SARS-COV-2 and Other mRNA Vaccines, from virus sequence availability. Case studies of three SARS-COV-2 mRNA vaccines for which field clinical efficacy data are available are examined and related to mRNA/LNP attributes where possible. The status of other SARS-COV-2 and non-SARS-COV-2 mRNA vaccines in clinical development and select future prospective innovations are reviewed.

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,Preparation of Synthetic mRNAs—Overview and Considerations,thetic mRNA is prepared in vitro via a cell-free enzymatic process. The aim of this chapter is to provide an overview of the enzymatic mRNA synthesis process and discuss the enzymatic components involved in the workflow. Furthermore, critical parameters that need to be considered for characterization of the synthetic mRNAs are also discussed.

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Nuclear Export of mRNAs with Disease Pathogenesis and Therapeutic Implications,teins in different cellular compartments. In this chapter, we describe how gene expression is controlled at the level of mRNA export from nucleus to the cytoplasm with implications for disease pathogenesis and therapeutic development.

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2197-9731 merging class of drugs.Provides a state-of-the-art overview This book focuses on the fundamentals and applications of messenger RNA (mRNA)-based therapeutics and discusses the strengths and key challenges of this emerging class of drugs. In the past 30 years, extensive research and technological dev

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Messenger RNA Therapeutics: Start of a New Era in Medicine,hetic mRNA platforms, production, purification; strategies to increase the stability and reduce the immunogenicity of therapeutic mRNA molecules; delivery methods of these mRNAs in vivo; and their applications, safety, and efficacy.

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Nonsequential Pre-mRNA Splicing: From Basic Understanding to Impacts on Splice-Manipulating Therapio study intron removal order and the occasions that they are best suited. Finally, we discuss how nonsequential splicing could facilitate or impede the development of splice-manipulating AOs and aspects to consider when analysing AO effectiveness.

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Advances in mRNA Delivery and Clinical Applications, vitro transcription (IVT), and, especially, delivery technology, the therapeutic mRNA got immense popularity. In this chapter, we described the latest advances in mRNA delivery systems as well as tried to figure out the development trend of delivery platforms and also summarized the applications of mRNA therapeutics.