Messenger RNA Therapeutics PDF Download

Author: Stefan Jurga
Publisher: Springer Nature
ISBN: 3031084152
Category : Medical
Languages : en
Pages : 451

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Book Description
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.

Author: Mouldy Sioud
Publisher: Humana Press
ISBN: 9781607616641
Category : Science
Languages : en
Pages : 527

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Book Description
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.

Author: Robert E. Rhoads
Publisher: Humana
ISBN: 9781493936236
Category : Medical
Languages : en
Pages : 0

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Book Description
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.

Author: Stefan Jurga
Publisher: Springer Nature
ISBN: 3030716120
Category : Science
Languages : en
Pages : 632

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Book Description
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.

Author: Masaki Ieda
Publisher: Springer
ISBN: 3319561065
Category : Medical
Languages : en
Pages : 277

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Book Description
This Volume of the series Cardiac and Vascular Biology offers a comprehensive and exciting, state-of-the-art work on the current options and potentials of cardiac regeneration and repair. Several techniques and approaches have been developed for heart failure repair: direct injection of cells, programming of scar tissue into functional myocardium, and tissue-engineered heart muscle support. The book introduces the rationale for these different approaches in cell-based heart regeneration and discusses the most important considerations for clinical translation. Expert authors discuss when, why, and how heart muscle can be salvaged. The book represents a valuable resource for stem cell researchers, cardiologists, bioengineers, and biomedical scientists studying cardiac function and regeneration.

Author: Sarfaraz K. Niazi
Publisher: CRC Press
ISBN: 1000788067
Category : Medical
Languages : en
Pages : 327

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Book Description
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.

Author:
Publisher: Elsevier
ISBN: 0443223157
Category : Science
Languages : en
Pages : 328

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Book Description
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

Author: Anya Hillery
Publisher: Elsevier
ISBN: 0443289352
Category : Medical
Languages : en
Pages : 318

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Book Description
mRNA Therapeutics: Foundations, Innovations and Clinical Applications aims to provide a comprehensive text that covers all aspects of mRNA therapeutics, from the foundational science that underpins this disruptive new drug class, through the scientific and technological breakthroughs crucial for therapeutic success, to the current clinical applications and the innovative advances driving future directions. The book begins with foundational knowledge covering mRNA biology, the immune system, and vaccines. The second section addresses the major challenges associated with mRNA as a therapeutic modality, and the molecular engineering innovations and delivery technologies that have allowed these hurdles to be largely overcome. The third section describes the current and future clinical applications of mRNA therapeutics that are transforming, or are poised to transform, medicine and health. This includes the use of mRNA vaccines for COVID-19 and other infectious diseases, as well as mRNA's role in revolutionizing cancer immunotherapy, covering immunostimulants, cancer vaccines including personalized neoantigen vaccines, and CAR T cell technologies. Additional chapters describe the use of mRNA therapeutics for protein replacement therapy and gene-editing, as well as newer mRNA constructs, including self-amplifying mRNA. The final section addresses the safety and regulatory considerations of mRNA therapeutics, along with broader cultural issues including vaccine hesitancy, global vaccine inequality, and pandemic preparedness. Currently, mRNA texts either provide personal accounts from key players involved in COVID-19 vaccine development, with limited scientific depth, or focus on highly specialized, more esoteric applications of mRNA in advanced molecular biology. This book aims to bridge this gap by providing a scientifically rigorous and wide-ranging exploration of mRNA's role in therapeutics. This pioneering textbook serves as a vital addition to the academic canon, providing an essential tool for the current and next generation of students, scientists, researchers and professionals in a wide variety of related disciplines including molecular biology, biomedical engineering, pharmaceutical science, oncology, and the health sciences. - 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

Author: Amanda L. Garner
Publisher: Springer
ISBN: 3319680919
Category : Science
Languages : en
Pages : 263

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Book Description
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.

Author: Peixuan Guo
Publisher: CRC Press
ISBN: 1466505664
Category : Medical
Languages : en
Pages : 614

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Book Description
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.