Messenger RNA therapeutics / Stefan Jurga, Jan Barciszewski, editors.
Jurga, Stefan, editor.; Barciszewski, Jan, editor.
2022
RM263
Available Online

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Title Messenger RNA therapeutics / Stefan Jurga, Jan Barciszewski, editors.
ISBN 9783031084157 (electronic bk.)
3031084152 (electronic bk.)
9783031084140
3031084144
DOI https://doi.org/10.1007/978-3-031-08415-7
Published Cham : Springer, [2022]
Copyright ©2022
Language English
Description 1 online resource (xii, 446 pages) : illustrations (chiefly color).
Other Standard Identifiers 10.1007/978-3-031-08415-7 doi
Call Number RM263
Dewey Decimal Classification 615.5/8
Summary 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 development in many areas have contributed to the emergence of in vitro transcribed mRNA as a therapeutic that has now reached clinical testing. Formulations that protect the mRNA from nucleases and accelerate its cellular uptake, combined with improvements to the mRNA molecules themselves, have been critical advancements for mRNAs to become viable therapeutics. Though once regarded as a serious impediment, the transient nature of mRNA technology is now considered a major advantage in making mRNA therapies safe and, ultimately, a potential game changer in the field of medicine. This new book in the RNA Technologies series provides a state-of-the-art overview on the emerging field of mRNA therapeutics covering essential strategies for formulation, delivery, and application. It also reviews the promising role in cancer immunotherapy, respiratory diseases, and chronic HBV infection and discusses RNA vaccines in light of the current COVID-19 pandemic. mRNA-based approaches have great potential to revolutionize molecular biology, cell biology, biomedical research, and medicine. Thus, this handbook is an essential resource for researchers in academia and industry contributing to the development of this new area of therapeutics.
Access Note Access limited to authorized users.
Source of Description Online resource; title from PDF title page (SpringerLink, viewed October 4, 2022).
Added Author Jurga, Stefan, editor.
Barciszewski, Jan, editor.
Series RNA technologies ; v. 13.
Available in Other Form Print version: 9783031084140
Linked Resources Online Access
Record Appears in Online Resources > Ebooks
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Intro
Introduction: IVT Messenger RNA in the Syringe
Contents
Roadmap to the Development of mRNA Therapeutics: From Molecule Design and Delivery Strategies to Manufacturing, Quality Control, and Regulatory Considerations
1 Introduction
2 mRNA Design Strategies
2.1 Reducing mRNA Immunogenicity
2.2 Optimizing Protein Expression
2.3 Optimizing mRNA Stability
3 Delivery Strategies for mRNA Therapeutics
3.1 Lipid Nanoparticles for mRNA Delivery
3.2 Other Materials for mRNA Delivery
3.3 mRNA Drug Product Route of Administration

4 Manufacturing of mRNA Drug Products
4.1 mRNA Drug Substance Manufacturing
4.2 mRNA Drug Product Manufacturing
5 Quality Control of mRNA Therapeutics
5.1 Drug Substance (mRNA) CQAs and Quality Control Strategy
5.2 Drug Product (mRNA-LNPs) CQAs and Quality Control Strategy
6 Conclusion and Future Directions
References
Messenger RNA for Prophylaxis
1 Introduction
2 mRNA Vaccines: Categories and Biological Function
3 Advances in mRNA Constructs
3.1 5' Capping
3.2 Untranslated Regions (UTRs)
3.3 Poly(A)-Tail

3.4 Nucleotide Modification and Codon Optimization
3.5 Purity
3.6 Self-Amplifying Specific Features
4 mRNA Carrier Technologies
4.1 Tropism and Uptake Efficiency
4.2 The Art of Endosomal Escape
4.3 Formulation
4.4 Stability
5 Conclusions
References
Messenger RNA Therapeutics: Start of a New Era in Medicine
1 Introduction
2 Production of IVT mRNA
3 Immunogenicity of IVT mRNA
4 Strategies to Increase the Stability and Reduce the Immunogenicity of IVT mRNA
4.1 Capping (m7GpppN or m7Gp3N)
4.2 Tailing
4.3 Untranslated Regions (UTRs)

4.4 Coding Region
5 Purification of Synthetic mRNA
6 Synthetic mRNA Platforms and their Features
6.1 Unmodified mRNA
6.2 Modified mRNA
6.3 Sequence-Optimized Unmodified mRNA
6.4 Replicon RNA
7 In Vivo Delivery Strategies of Exogenous mRNA
7.1 Delivery of Naked mRNA
7.2 Cationic Liposome-Mediated or Cationic Nanoemulsion (CNE)-based RNA Transfection
7.3 Peptide-based Delivery
7.4 Electroporation and Nucleoporation
7.5 Gene Gun-Mediated Delivery of mRNA
7.6 Use of Polymer Nanomaterials
7.7 Virus-like Replicon Particle (VRP)-based Delivery of mRNA

7.8 Other Lesser-Known Methods
8 Applications of mRNA Therapeutics
8.1 mRNA as a Therapeutic Agent for Replacement of Defective Protein within the Cell
8.2 mRNA as Vaccines Against Cancer
8.3 Dendritic Cell (DC) Vaccines
8.4 mRNA Vaccines in Prevention of Diseases
8.5 mRNA-Mediated Genome Editing
8.6 Generation of Induced Pluripotent Stem Cells (iPSCs) using mRNA
9 Safety of mRNA Therapeutics
9.1 The Safety Concern Over the Use of Non-Natural Nucleotides/Nucleosides in IVT mRNA
9.2 Safety Considerations Regarding the Encoded Protein
10 Conclusions
References

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