Download or read book Messenger RNA Therapeutics written by Stefan Jurga and published by Springer Nature. This book was released on 2022-09-20 with total page 451 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book MRNA Based Therapeutics written by Fernando Aranda Vega and published by Elsevier. This book was released on 2022-09-07 with total page 326 pages. Available in PDF, EPUB and Kindle. Book excerpt: MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review of Cell and Molecular Biology serials Updated release includes the latest information on MRNA-Based Therapeutics

Download or read book Synthetic mRNA written by Robert E. Rhoads and published by Humana. This book was released on 2016-05-29 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume presents detailed laboratory protocols for in vitro synthesis of mRNA with favorable properties, its introduction into cells by a variety of techniques, and the measurement of physiological and clinical consequences such as protein replacement and cancer immunotherapy. Synthetic techniques are described for structural features in mRNA that provide investigational tools such as fluorescence emission, click chemistry, photo-chemical crosslinking, and that produce mRNA with increased stability in the cell, increased translational efficiency, and reduced activation of the innate immune response. Protocols are described for clinical applications such as large-scale transfection of dendritic cells, production of GMP-grade mRNA, redirecting T cell specificity, and use of molecular adjuvants for RNA vaccines. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Synthetic mRNA: Production, Introduction into Cells, and Physiological Consequences is a valuable and cutting-edge resource for both laboratory investigators and clinicians interested in this powerful and rapidly evolving technology.

Download or read book RNA Therapeutics written by Mouldy Sioud and published by Humana Press. This book was released on 2011-03-04 with total page 527 pages. Available in PDF, EPUB and Kindle. Book excerpt: Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular BiologyTM series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.

Download or read book RNA Therapeutics written by Mouldy Sioud and published by Humana Press. This book was released on 2011-03-04 with total page 527 pages. Available in PDF, EPUB and Kindle. Book excerpt: Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular BiologyTM series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.

Download or read book mRNA Therapeutics written by Sarfaraz K. Niazi and published by CRC Press. This book was released on 2022-12-06 with total page 327 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book is the most comprehensive and complete treatise on nucleic acid therapeutic products, including mRNA vaccines, their manufacturing, formulations, and testing for safety and efficacy. Details include cGMP-compliant manufacturing and regulatory filing steps. A new concept of “biosimilar” mRNA vaccine is presented to secure fast approval of copies of mRNA vaccines. Projections of financial plans to establish RNA manufacturing facilities are provided, along with details of supply chain management. Finally, the future of nucleic acid products in gene therapy and other newer applications is presented, along with a perspective that all new vaccines will be the nucleic acid type that will further provide first-time prevention of autoimmune disorders. It is projected that both big pharma and start-ups will enter this field, and we can expect significant additions to our drug armamentarium soon.

Download or read book RNA Therapeutics Part A written by and published by Elsevier. This book was released on 2024-02-13 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA Therapeutics, Part A, Volume 203 presents the latest on a new class of medication based on RNA molecules. The book includes timely chapters that focus on An introduction to RNA therapeutics and potentials, The development and technologies of RNA therapeutics, Types of RNA therapeutics, Molecular mechanisms of RNA therapeutics, IT, and AI applications in RNA therapeutics, The application of data sciences and bioinformatics in RNA therapeutics, RNA therapeutics history and future perspectives, Recent applications of RNA therapeutic in clinics, Advantages and disadvantages of RNA therapeutics, RNA therapeutics for neurological disease, RNA therapeutics for metabolic disorders, and RNA therapeutics for metabolic disorders. Discusses the history of RNA therapeutics, along with future perspectives Presents the processes of RNA therapeutical developments Covers recent applications of RNA therapeutics in clinics Provides the advantages and disadvantages of RNA therapeutics

Download or read book The Marathon of the Messenger written by Jérôme Lemonnier and published by Springer Nature. This book was released on 2023-12-03 with total page 208 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Covid-19 pandemic changed the world. Indeed a real race took place worldwide between SARS-CoV-2 on the one hand and researchers on the other – especially those specializing in messenger RNA vaccines. Four years after its emergence, the pandemic is not over, but some decisive battles have been won, thanks to the great success of mRNA vaccines. The Marathon of The Messenger presents the history of these mRNA vaccines, combining a scientific background with historical and economic perspectives. It appears that an important page in the history of these new vaccines was written in Europe, thanks to the crucial work of German and French scientists; this effort began in 1993 and continues to this day. In the face of a prevailing single-mindedness, these researchers pushed through a new therapeutic concept and defined the biotechnological keys that would open the way to the production of therapeutic messenger RNA in the fight against cancer and viral infections. Written for a broad audience and accompanied by humorous cartoons, this book will appeal to anyone looking for scientific and historical answers about mRNA vaccines. Readers will discover not only the technical and scientific knowledge of how these vaccines work, but also the economic levers that were necessary to create this technology. This book has been written in collaboration with Dr. Steve Pascolo, former director of CureVac, and the RNA messenger expert Professor Chantal Pichon. It also features a preface by Dr. Pierre Meulien, former director of the European Union public-private partnership Innovative Medicines Initiative (IMI).

Download or read book MRNA Based Therapeutics written by and published by Academic Press. This book was released on 2022-09-03 with total page 328 pages. Available in PDF, EPUB and Kindle. Book excerpt: MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review of Cell and Molecular Biology serials Updated release includes the latest information on MRNA-Based Therapeutics

Download or read book mRNA Therapeutics written by Anya Hillery and published by Elsevier. This book was released on 2024-09-16 with total page 318 pages. Available in PDF, EPUB and Kindle. Book excerpt: mRNA Therapeutics: Foundations, Innovations, and Clinical Applications summarizes the scientific development of mRNA vaccines that have revolutionized the way we treat disease. This approach has the potential to treat a wide range of diseases, including COVID-19, cancer, and infectious diseases. The first section of the book introduces mRNA vaccines, including their history, mechanism of action, and potential applications. The second section delves into the science of mRNA vaccines, covering topics such as the structure of mRNA, the delivery of mRNA to cells, and the immune response to mRNA vaccines. The third section discusses the future of mRNA technology, exploring potential applications such as cancer therapy, prevention of infectious diseases, and replacement of missing or damaged proteins. This is a valuable resource for scientists, researchers, and clinicians who are interested in the latest developments in mRNA technology. It is also a useful reference for students and professionals who are working in the fields of biotechnology, pharmaceutical science, and vaccine development. Focuses on the science of mRNA, covering the development, modus operandi, platform manufacturing technology, safety, and efficacy of this treatment modality Provides the mRNA technology fit with the wider context of vaccinology, virology, oncology, biotechnology, as well as manufacturing and regulatory science Offers an understanding of the recent paradigm shift in the way we treat disease

Download or read book RNA Therapeutics written by Paloma H. Giangrande and published by Academic Press. This book was released on 2022-04-10 with total page 474 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an emphasis on the increasingly impactful areas of gene therapy, oligonucleotide therapeutics, gene editing and delivery. International leaders in the field examine RNA-based therapeutics tools that have been developed to-date to modulate cellular processes such as transcription, translation and protein function. Approved RNA-based therapies and lessons learned from failed therapies are discussed in-depth, as are evolving advances in RNA biochemical analysis, and similar advances that are enabling clinical application of RNA-based therapies. Later sections discuss delivery technologies, remaining hurdles in research and translation, the therapy development process from the lab to the clinic, and novel RNA-based therapies currently in development. Features leading experts in the field of RNA therapeutics, spanning all classes of RNA therapies Provides a detailed examination of approved RNA therapies and lessons learned from failed therapeutics Covers all aspects of therapeutic discovery and preclinical development, as well as clinical translation, manufacturing and regulatory aspects

Download or read book RNA Therapeutics written by Amanda L. Garner and published by Springer. This book was released on 2017-09-26 with total page 263 pages. Available in PDF, EPUB and Kindle. Book excerpt: Medicinal chemistry is both science and art. The science of medicinal chemistry offers mankind one of its best hopes for improving the quality of life. The art of medicinal chemistry continues to challenge its practitioners with the need for both intuition and experience to discover new drugs. Hence sharing the experience of drug research is uniquely beneficial to the field of medicinal chemistry. Drug research requires interdisciplinary team-work at the interface between chemistry, biology and medicine. Therefore, the topic-related series Topics in Medicinal Chemistry covers all relevant aspects of drug research, e.g. pathobiochemistry of diseases, identification and validation of (emerging) drug targets, structural biology, drugability of targets, drug design approaches, chemogenomics, synthetic chemistry including combinatorial methods, bioorganic chemistry, natural compounds, high-throughput screening, pharmacological in vitro and in vivo investigations, drug-receptor interactions on the molecular level, structure-activity relationships, drug absorption, distribution, metabolism, elimination, toxicology and pharmacogenomics. In general, special volumes are edited by well known guest editors.

Download or read book The Marathon of the Messenger written by Jérôme Lemonnier and published by Springer. This book was released on 2023-11-09 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Covid-19 pandemic changed the world. Indeed a real race took place worldwide between SARS-CoV-2 on the one hand and researchers on the other – especially those specializing in messenger RNA vaccines. Four years after its emergence, the pandemic is not over, but some decisive battles have been won, thanks to the great success of mRNA vaccines. The Marathon of The Messenger presents the history of these mRNA vaccines, combining a scientific background with historical and economic perspectives. It appears that an important page in the history of these new vaccines was written in Europe, thanks to the crucial work of German and French scientists; this effort began in 1993 and continues to this day. In the face of a prevailing single-mindedness, these researchers pushed through a new therapeutic concept and defined the biotechnological keys that would open the way to the production of therapeutic messenger RNA in the fight against cancer and viral infections. Written for a broad audience and accompanied by humorous cartoons, this book will appeal to anyone looking for scientific and historical answers about mRNA vaccines. Readers will discover not only the technical and scientific knowledge of how these vaccines work, but also the economic levers that were necessary to create this technology. This book has been written in collaboration with Dr. Steve Pascolo, former director of CureVac, and the RNA messenger expert Professor Chantal Pichon. It also features a preface by Dr. Pierre Meulien, former director of the European Union public-private partnership Innovative Medicines Initiative (IMI).

Download or read book Lipid Nanoparticle messenger RNA Formulations Against Infections written by Xucheng Hou and published by . This book was released on 2022 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: The rapid development and successful application of messenger RNA (mRNA) vaccines to ameliorate the coronavirus disease 2019 (COVID-19) pandemic highlight the potential of mRNA therapeutics to prevent and treat infectious diseases. To further facilitate the clinical translation of mRNA therapeutics, additional efforts are required to optimize targeted delivery systems, mRNA stability, manufacturing costs, and applications beyond severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) vaccines. The objective of this dissertation is to develop novel lipid nanoparticle (LNP)-mRNA formulations against bacterial and viral infections via synthesis of effective delivery materials to engineer macrophages, de novo design of mRNA structures to improve the stability and translational efficiency, and optimization of long-term storage conditions of LNP-mRNA formulations. Chapter 1 briefly reviews the strategies for optimizing mRNA structures, the representative LNPs for mRNA delivery, and the applications of LNP-mRNA formulations in infectious diseases. Chapter 2 demonstrates that the adoptive transfer of macrophages pre-storing antimicrobial peptides in their lysosomes (MACs) can be used to treat sepsis induced by multidrug-resistant bacteria. The MACs were constructed by ex vivo engineering with vitamin C LNPs encapsulating antimicrobial peptide linked cathepsin B (AMP-CatB) mRNA. The AMP-CatB mRNA enabled the accumulation of AMPs in macrophage lysosomes, a crucial organelle for bactericidal activities. In vivo data revealed that the adoptive MAC transfer reduced bacterial burden, resulting in the complete recovery of septic mice with immunosuppression. Chapter 3 describes the development of a COVID-19 vaccine candidate with optimized untranslated regions (UTRs) in mRNA. After bioinformatics analysis and de novo design of the UTRs, the optimal combination of 5’ and 3’ UTRs was identified and named as NASAR, which increased five to ten-fold mRNA translation compared to wild type UTRs. Next, a COVID-19 vaccine candidate was developed by formulating NASAR mRNA encoding SARS-CoV-2 antigens with N1, N3, N5-tris(3-(didodecylamino)propyl)benzene-1,3,5-tricarboxamide (TT3) LNPs. After a prime-boost vaccination, the vaccine induced strong spike-specific antibody responses in animal models. Chapter 4 investigates the conditions for long-term storage of LNP-mRNA formulations. The stability of TT3 LNPs was evaluated under freezing or lyophilization conditions with a series of concentrations of cryoprotectants (sucrose, trehalose, or mannitol). Through evaluating the in vitro and in vivo mRNA delivery efficacy, we identified that the supplement with 5% (w/v) sucrose or trehalose to TT3 LNPs maintained the mRNA delivery efficacy for three months under the liquid nitrogen storage condition. Collectively, these results demonstrate that LNP-mRNA formulations are promising tools for preventing and treating infections with clinical transferability.

Download or read book Epitranscriptomics written by Stefan Jurga and published by Springer Nature. This book was released on 2021-07-28 with total page 632 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book reviews a novel and exciting field of cellular and molecular biology called epitranscriptomics, which focuses on changes in an organism’s cells resulting from the posttranscriptional modification of cellular RNA. RNA-binding proteins (RBPs) play a crucial role in these posttranscriptional modifications and also support several cellular processes necessary for maintaining RNA homeostasis. Exploring the mechanisms underlying RNA modifications and RBP function is an emerging area of biomedical research, taking the study of gene regulation a step beyond epigenetics. This book reveals that the RNA molecule is not just an information-carrying molecule with some secondary structures. Accordingly, how RNA is modified, regulated, packaged, and controlled is an important aspect. Leading experts address questions such as where the over 170 distinct posttranscriptional RNA modifications are located on the genome, what percentage of mRNAs and noncoding RNAs these modifications include, and how an RNA modification impacts a person’s biology. In closing, the book reviews the role of RNA modifications and RBPs in a variety of diseases and their pathogenesis. Addressing some of the most exciting challenges in epitranscriptomics, this book provides a valuable and engaging resource for researchers in academia and industry studying the phenomena of RNA modification.

Download or read book RNA Therapeutics Part B written by and published by Elsevier. This book was released on 2024-03-06 with total page 360 pages. Available in PDF, EPUB and Kindle. Book excerpt: RNA Therapeutics, Part B, Volume 204 updates on a new class of medication based on RNA molecules, with chapters on a variety of topics, including RNA therapeutics for cardiovascular disorders, RNA therapeutics for kidney injury, RNA therapeutics for beta-thalassemia, RNA therapeutics for infectious diseases, RNA therapeutics for reproductive diseases, Potential of RNA and transcriptomic data to drive drug repurposing for various diseases, RNA therapeutics for glioma/glioblastoma, RNA therapeutics for regenerative medicine, Advances in the liposome-based delivery systems for RNA therapeutics in cancer, Advances in the polymeric nanoparticulate delivery systems for RNA therapeutics in cancer, and more. Covers RNA therapeutics' history and future perspectives Discusses the processes of RNA therapeutical development Presents recent applications of RNA therapeutic in clinics Provides the advantages and disadvantages of RNA therapeutics

Download or read book RNA Nanotechnology and Therapeutics written by Peixuan Guo and published by CRC Press. This book was released on 2013-07-09 with total page 614 pages. Available in PDF, EPUB and Kindle. Book excerpt: Interest in RNA nanotechnology has increased in recent years as recognition of its potential for applications in nanomedicine has grown. Edited by the world's foremost experts in nanomedicine, this comprehensive, state-of-the-art reference details the latest research developments and challenges in the biophysical and single molecule approaches in RNA nanotechnology. In addition, the text also provides in-depth discussions of RNA structure for nanoparticle construction, RNA computation and modeling, single molecule imaging of RNA, RNA nanoparticle assembly, RNA nanoparticles in therapeutics, RNA chemistry for nanoparticle synthesis, and conjugation and labeling.