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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.
Auteur
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 Poznan (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 Poznan, Poland and at the Institute of Bioorganic Chemistry of the Polish Academy of Sciences, Poznan, 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.
Contenu
The Democratization of RNA Therapeutics.- Supramolecular Strategies for mRNA Delivery.- In Vitro-Transcribed mRNAs as a New Generation of Therapeutics in the Dawn of 21st Century: Exploitation of Peptides as Carriers for their Intracellular Delivery.- Effective Delivery of mRNA Therapeutics and Vaccines Using Lipid Nanoparticles.- Messenger RNA Therapeutics.- Formulation, Delivery and Application of mRNA Therapeutics In Respiratory Diseases.- Clinical Applications of mRNA Formulated In Lipid Nanoparticles.- Preparation of Messenger RNA Loaded Nanomedicine Applied on Tissue Engineering And Regenerative Medicine.- Lipid Nanoparticle-Mediated Delivery of mRNA Therapeutics: Immune Activation By Ionizable Cationic Lipids.- Adjuvants, the Elephant in the Room for RNA Vaccines.- Addressing Skin with Modified mRNA Constructs for Novel Therapies.- mRNA-Based Cancer Immunotherapies.- From Bench to Bedside: The Journey of an mRNA Drug Candidate Into a Medicine.- Synthetic mRNA Gene Therapies for Chronic HBV Infection.- Delivery Vehicles for Self-Amplifying RNA.- Delivery Technologies of mRNA Vaccines.- Nanosystems: The Key in Formulations of mRNA for Several Pathologies.- Messenger RNA Nanoformulation For Cancer Vaccine.- Preparation of Synthetic mRNAs for In Vivo Applications Overview and Considerations.- Messenger RNA for Prophylaxis.- Nuclear Export of mRNA with Disease Pathogenesis and Therapeutic Potentials.- Messenger RNA Therapeutics: Start of a New Era in Medicine.- mRNA Delivery Technologies for Therapeutic Applications. <p
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