Messenger RNA Therapeutics

1. Front Matter

   Pages i-xii

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2. Roadmap to the Development of mRNA Therapeutics: From Molecule Design and Delivery Strategies to Manufacturing, Quality Control, and Regulatory Considerations

   • Randall A. Meyer, Sara Trabulo, Julie A. Douthwaite, Jose Luis Santos

   Pages 1-16

3. Messenger RNA for Prophylaxis

   • Nicholas Jackson

   Pages 17-40

4. Messenger RNA Therapeutics: Start of a New Era in Medicine

   • Saloni Jain, Abhilash J. George, Vasu Sharma, Gagandeep Singh, Vandana Gupta

   Pages 41-71

5. Hospital-Based RNA Therapeutics

   • Tulsi Ram Damase, Roman Sukhovershin, Min Zhang, Daniel L. Kiss, John P. Cooke

   Pages 73-92

6. Medical Use of mRNA-Based Directed Gene Delivery

   • A. C. Matin, Alexis Forterre

   Pages 93-112

7. SARS-COV-2 and Other mRNA Vaccines

   • Nicholas Jackson

   Pages 113-138

8. Pulmonary Delivery of Messenger RNA (mRNA) Therapeutics for Respiratory Diseases

   • Yingshan Qiu, Michael Yee-Tak Chow, Jenny Ka-Wing Lam

   Pages 139-156

9. Synthetic mRNA Gene Therapies and Hepatotropic Non-viral Vectors for the Treatment of Chronic HBV Infections

   • Dylan Kairuz, Prashika Singh, Tiffany Smith, Patrick Arbuthnot, Abdullah Ely, Kristie Bloom

   Pages 157-179

10. Preparation of Synthetic mRNAs—Overview and Considerations

    • Siu-Hong Chan, Bijoyita Roy

    Pages 181-207

11. In Vitro-Transcribed mRNAs as a New Generation of Therapeutics in the Dawn of Twenty-First Century: Exploitation of Peptides as Carriers for Their Intracellular Delivery

    • A. N. Miliotou, I. S. Pappas, I. S. Vizirianakis, L. C. Papadopoulou

    Pages 209-235

12. Lipid Nanoparticle-Mediated Delivery of Therapeutic and Prophylactic mRNA: Immune Activation by Ionizable Cationic Lipids

    • Melike Ongun, Abhijeet Girish Lokras, Camilla Foged, Aneesh Thakur

    Pages 237-255

13. Adjuvants, the Elephant in the Room for RNA Vaccines

    • Sigrid D’haese, Sabine den Roover, Joeri L. Aerts

    Pages 257-276

14. Advances in mRNA Delivery and Clinical Applications

    • Bo Hu, Abid Hussain, Qing Liu, Yuhua Weng, Yuanyu Huang

    Pages 277-305

15. Lipid Nanoparticles to Harness the Therapeutic Potential of mRNA for Cancer Treatment

    • Maria L. Guevara, Francesca Persano, Stefano Persano

    Pages 307-336

16. RNA/Polymer-Based Supramolecular Approaches for mRNA Delivery

    • Eger Boonstra, Satoshi Uchida, Horacio Cabral

    Pages 337-354

17. Delivery Vehicles for Self-amplifying RNA

    • Nuthan Vikas Bathula, Petya Popova, Anna Blakney

    Pages 355-370

18. Nuclear Export of mRNAs with Disease Pathogenesis and Therapeutic Implications

    • Shalini Guha, Priyanka Barman, Aruniti Manawa, Sukesh R. Bhaumik

    Pages 371-395

19. Preparation of Messenger RNA-Loaded Nanomedicine Applied on Tissue Engineering and Regenerative Medicine

    • Hsi-Kai Tsou, Cheng-Chung Chang, Tomoji Maeda, Chin-Yu Lin

    Pages 397-428

20. Nonsequential Pre-mRNA Splicing: From Basic Understanding to Impacts on Splice-Manipulating Therapies

    • Kristin A. Ham, Steve D. Wilton, May T. Aung-Htut

    Pages 429-446

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.

• mRNA therapeutics
• RNA vaccines
• SARS-CoV-2
• Lipid nanoparticles
• Adjuvants
• Cancer Immunotherapy
• Self-amplifying RNA
• mRNA delivery technologies
• Nanosystems
• Immunogenicity

• NanoBioMedical Center, Adam Mickiewicz University, Poznań, Poland

  Stefan Jurga, Jan Barciszewski

Professor Stefan Jurga conducts interdisciplinary scientific research in the field of nanoscience and nanotechnology, covering the physical, chemical, biological and medical sciences. He has authored over 270 publications in the interdisciplinary database Journal Citation Reports. He has served as Visiting Professor at, e.g., Cornell University, North Carolina State University, and the University of South Africa in Pretoria. He was a laureate of the Alexander von Humboldt Foundation and a scholarship holder at the Max Planck Institute for Polymer Research in Mainz (Germany). He has also worked at the University of Illinois at Urbana Champaign, at the Józef Stefan Institute in Ljubljana, and at many other international scientific institutions. He has served as Vice-Rector and Rector of the Adam Mickiewicz University Poznań (AMU) and also created the interdisciplinary NanoBioMedical Center at the AMU, which he has been the director of since 2010. From 2005 to 2007, he was appointed as the Under Secretary and Secretary of State for research and higher education at the Ministry of Education and Science and the Ministry of Science and Higher Education.

Jan Barciszewski is Professor at Adam Mickiewicz University (AMU) in Poznań, Poland and at the Institute of Bioorganic Chemistry of the Polish Academy of Sciences, Poznań, Poland where he has worked since 1974. He studied organic chemistry at the AMU. During his Ph.D. studies, he worked on the structure and function of modified bases and nucleoside sequences of plant phenylalanine specific transfer ribonucleic acid (tRNA), including cytokinins. He was subsequently granted Doctor of Science degree for his work on the properties of plant tRNAs and aminoacyl-tRNA synthetases. In the 1990s, he began working on the diagnosis and therapy of brain tumors. He developed a new method for the transformation of plant mitochondria based on catalytic RNAs and is currently involved in studies on a new type of catalytic RNAs (enantiomeric ribozymes) for efficient RNA target cleavage in vivo, as well as the search for new anti-aging agents.

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