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.
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.
RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with 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 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
The enormous potential of siRNA as a therapeutic has led to an explosion of interest from the scientific community. There has been intense interest from Big Pharma to capitalise on this new technology but the fact remains that delivery is a key determinant in realizing the full clinical potential of RNA interference. There is an urgent need for better delivery methods to take this technology forward. This book addresses the role of different RNAi molecules in cellular processes as rational for diagnostic and therapeutic approaches. This book will cover RNAi therapeutic design to optimize siRNA potency and reduce off-target effects and current delivery technologies to overcome both intracellular and extracellular barriers. The reader will gain an insight into RNA interference from the cellular mechanisms to screening to siRNA design right through to diagnostic and therapeutic applications.
|Author||: Anna Zampetaki,Lars Maegdefessel|
|Publisher||: Frontiers Media SA|
|Release Date||: 2020-07-22|
|ISBN 10||: 2889638820|
|Pages||: 329 pages|
Cooperation has become the leading strategy adopted by business and other organizations. It is taking on new forms that are adapted to changing market expectations and technological possibilities in the rapidly evolving business environment. This new edition of Cooperative Strategy provides a comprehensive view of the practical and theoretical literature concerning cooperative strategies, and the alliance and network organizational forms that are the enablers of these strategies. It takes the reader through the stages of developing a cooperative alliance, from choosing a cooperative form and selecting partners, to establishing an alliance and managing the process of cooperation. It examines cooperative strategies in different sectors as well as internationally, and discusses performance criteria and evolution of cooperation over time. With insights from internationally recognized experts on cooperative strategy, this book presents extensive research on the topic while also addressing practical issues of alliance management. Echoing the words of the famous social psychologist Kurt Lewin that 'there is nothing so practical as a good theory', the authors provide a sound understanding of the theory and research on cooperative strategy so as to inform its practice. In this respect, this new edition follows its predecessor as an essential resource for both students and managers alike.
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 R
The ‘RNA world’ hypothesis that proposed RNA molecules as the first form of genetic material was put forwarded in the late 1980s but got impetus only recently when high-throughput sequencing technologies began unearthing new types of non-coding RNA (ncRNA) transcripts in higher eukaryotes. Till then, research on ncRNAs were primarily confined to transfer RNAs and, ribosomal RNAs, which act as the messengers of the protein synthesis and allow translation of genetic information encoded by DNA into proteins. In recent years, the integration of high-throughput genomic technologies with molecular biology and omics sciences have revolutionized the fields of ncRNA research by identifying the hidden treasures of several new types of ncRNAs encoded in the genomes of several organisms and decrypting their versatile roles in gene expression and epigenetics. Among these, two small endogenous ncRNAs, namely microRNAs (miRNAs) and piwi-interacting RNAs (piRNAs) that drive argonaute (AGO) family of proteins namely Ago and Piwi respectively and silence the expression of genes have geared up molecular and disease biology research in recent years. Both miRNAs and piRNAs are expressed in higher eukaryotes, including human and act as cellular rheostats by regulating the expression of significant fraction of genes encoded in the genomes. The aberrant expressions of these small ncRNAs within the cells cause various abnormalities and diseases including cancer. Manipulating their aberrant expression or function can serve as potential novel diagnostic/prognostic biomarkers and bring in new therapeutic strategies for multiple human diseases. This can be further translated from bench-side to clinic for improving human health. This book captures the essence of the pioneering work of some of the world’s leading researchers showcasing the scientific excitements surrounding the evolving regulatory roles of miRNAs and piRNAs highlighting their potential towards the diagnosis and therapeutics of various diseases. The book is geared towards scientists, students, and will particularly appeal to active investigators in RNA biology, molecular biology, cancer research as well as clinicians and will provide them a comprehensive view of recent discoveries and research progresses to utilize miRNAs, piRNAs and their interacting proteins, Ago and Piwi for diagnosis, prognosis and therapeutics of diseases. Provides a unified cutting-edge resource for both miRNAs and piRNAs, two promising AGO-clade small ncRNAs, their functions and potential applications Showcase high-throughput technologies and other approaches for discovery of these small ncRNAs and their targets Unveils the diverse molecular mechanisms by which miRNAs and piRNAs regulate gene expression in animal cells Showcase recent discoveries on involvement of Argonaute and Piwi proteins in different biological processes and diseases as well as their possible use in diagnosis Report breakthroughs in the use of small ncRNAs for diagnosis and personalized therapy
RNA technologies are the driving forces of modern medicine and biotechnology. They combine the fields of biochemistry, chemistry, molecular biology, cell biology, physics, nanotechnology and bioinformatics. The combination of these topics is set to revolutionize the medicine of tomorrow. After more than 15 years of extensive research in the field of RNA technologies, the first therapeutics are ready to reach the first patients. Thus we are witnessing the birth of a very exciting time in the development of molecular medicine, which will be based on the methods of RNA technologies. This volume is the first of a series. It covers various aspects of RNA interference and microRNAs, although antisense RNA applications, hammerhead ribozyme structure and function as well as non-coding RNAs are also discussed. The authors are internationally highly respected experts in the field of RNA technologies.
In the past few decades there has been incredible growth in "bionano"-related research, which has been accompanied by numerous publications in this field. Although various compilations address topics related to deoxyribonucleic acid (DNA) and protein, there are few books that focus on determining the structure of ribonucleic acid (RNA) and using RNA as building blocks to construct nanoarchitectures for biomedical and healthcare applications. RNA Nanotechnology is a comprehensive volume that details both the traditional approaches and the latest developments in the field of RNA-related technology. This book targets a wide audience: a broad introduction provides a solid academic background for students, researchers, and scientists who are unfamiliar with the subject, while the in-depth descriptions and discussions are useful for advanced professionals. The book opens with reviews on the basic aspects of RNA biology, computational approaches for predicting RNA structures, and traditional and emerging experimental approaches for probing RNA structures. This section is followed by explorations of the latest research and discoveries in RNA nanotechnology, including the design and construction of RNA-based nanostructures. The final segment of the book includes descriptions and discussions of the potential biological and therapeutic applications of small RNA molecules, such as small/short interfering RNAs (siRNAs), microRNAs (miRNAs), RNA aptamers, and ribozymes.
A flurry of recent research on the role of the RNA/DNA-binding proteins TDP-43 and FUS as well as a dozen other factors (e.g., C9ORF72 and profilin) has led to a new paradigm in our understanding of the pathobiology of the motor neuron disease, Amyotrophic Lateral Sclerosis (ALS). How these factors trigger neuromuscular dysfunction is critical for developing more effective ALS therapeutics. The ‘gain-of-toxicity’ or ‘loss-of-function’ of these etiological factors is a key question. Recent studies on the imbalance in genome damage versus repair have opened avenues for potential DNA repair-based therapeutics. This book highlights emerging science in the area of ALS and discusses key approaches and mechanisms essential for developing a cure for ALS.
RNA-based Regulation in Human Health and Disease offers an in-depth exploration of RNA mediated genome regulation at different hierarchies. Beginning with multitude of canonical and non-canonical RNA populations, especially noncoding RNA in human physiology and evolution, further sections examine the various classes of RNAs (from small to large noncoding and extracellular RNAs), functional categories of RNA regulation (RNA-binding proteins, alternative splicing, RNA editing, antisense transcripts and RNA G-quadruplexes), dynamic aspects of RNA regulation modulating physiological homeostasis (aging), role of RNA beyond humans, tools and technologies for RNA research (wet lab and computational) and future prospects for RNA-based diagnostics and therapeutics. One of the core strengths of the book includes spectrum of disease-specific chapters from experts in the field highlighting RNA-based regulation in metabolic & neurodegenerative disorders, cancer, inflammatory disease, viral and bacterial infections. We hope the book helps researchers, students and clinicians appreciate the role of RNA-based regulation in genome regulation, aiding the development of useful biomarkers for prognosis, diagnosis, and novel RNA-based therapeutics. Comprehensive information of non-canonical RNA-based genome regulation modulating human health and disease Defines RNA classes with special emphasis on unexplored world of noncoding RNA at different hierarchies Disease specific role of RNA - causal, prognostic, diagnostic and therapeutic Features contributions from leading experts in the field