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.

Technologies collectively called omics enable simultaneous measurement of an enormous number of biomolecules; for example, genomics investigates thousands of DNA sequences, and proteomics examines large numbers of proteins. Scientists are using these technologies to develop innovative tests to detect disease and to predict a patient's likelihood of responding to specific drugs. Following a recent case involving premature use of omics-based tests in cancer clinical trials at Duke University, the NCI requested that the IOM establish a committee to recommend ways to strengthen omics-based test development and evaluation. This report identifies best practices to enhance development, evaluation, and translation of omics-based tests while simultaneously reinforcing steps to ensure that these tests are appropriately assessed for scientific validity before they are used to guide patient treatment in clinical trials.

This volume explores the uses of RNAi and CRISPR interferences as a general method for inhibiting gene expression, with focus on their biological functions, design, chemical modifications, delivery, and preclinical/clinical applications. In addition to relevant backgrounds, the chapters in this book discuss simple and accurate protocols dealing with the latest advances in RNAi and CRISPR applications and look at how these methods differ from one another. 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, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Comprehensive and cutting-edge, RNA Interference and CRISPR Technologies: Technical Advances and New Therapeutic Opportunities is a valuable resource for any scientist, teachers, graduate student, postdoc, and clinician interested in this field. This book also benefits anyone in research and development in biotech and pharmaceutical companies who want to understand more about these technologies, and their applications in biology and medicine.

RNAi technology is used for large-scale screens that systematically shut down each gene in the cell, which can help identify the components necessary for a particular cellular process or an event such as cell division. Exploitation of the pathway is also a promising tool in biotechnology and medicine. Introducing new technology in the study of RNA

Maximizing the potential of RNA interference in functional genomics - as well as in the development of therapeutics - continues to be at the forefront of biomedical research. Unlike journal articles, Gene Silencing by RNA Interference: Technology and Application combines essential background to the RNAi field with practical techniques designed by renowned researchers to provide the most diverse and in-depth examination of the subject yet. This book describes methods and protocols for gene silencing and RNA interference. Each chapter provides necessary background to the subject and then gives detailed methods in easy-to-follow steps, along with troubleshooting hints and tips. Following a general and historical introduction, chapters two through eight focus on technical details of the various methods of siRNA design, chemical and enzymatic production, plasmid- and virus-mediated intracellular expression, and other RNAi tools. Chapter nine discusses the exogenous delivery of siRNAs into cells, and the final chapters of the book detail the application of RNAi to dissect gene function in a number of biological systems, including cell-free systems, cultured cells, and whole organisms. From the design and production of RNAi tools to their applications, Gene Silencing by RNA Interference: Technology and Application is the first to present the entire spectrum of activity, design, production, and delivery of RNAi reagents, providing a welcome guide for both academic and commercial use.

This book offers a unique and comprehensive overview of key RNA-based technologies, as well as their development and applications for the functional genomics of plant coding and non-coding genes. It focuses on the latest as well as classical RNA-based techniques used for studies on small RNAs, long non-coding RNAs and protein-coding genes. These techniques chiefly focus on target mimics (TMs) and short tandem target mimics (STTMs) for small RNAs, and artificial microRNAs (amiRNAs), RNA interference (RNAi) and CRISPR/Cas for genes. Furthermore, the book discusses the latest trends in the field and various modifications of the above-mentioned approaches, and explores how these RNA-based technologies have been developed, applied and validated as essential technologies in plant functional genomics. RNA-based technologies, their mechanisms of action, their advantages and disadvantages, and insights into the further development and applications of these technologies in plants are discussed. These techniques will enable the users to functionally characterize genes and small RNAs through silencing, overexpression and editing. Gathering contributions by globally respected experts, the book will appeal to students, teachers and scientists in academia and industry who are interested in horticulture, genetics, pathology, entomology, physiology, molecular genetics and breeding, in vitro culture & genetic engineering, and functional genomics.

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.

In this book the authors review the field and explore the potential role of RNAi and other RNA technologies in cardiovascular medicine and research. They highlight the impressive recent progress but also the hurdles that still must be overcome before this promising technology is finally ready for translation and clinical use.

Recent progress in high-throughput technologies and genome wide transcriptome studies have lead to a significant scientific milestone of discovering non-coding RNAs (ncRNAs) which spans through a major portion of the genome. These RNAs most often act as riboregulators, and actively participate in the regulation of important cellular functions at the transcriptional and/or post-transcriptional levels rather than simply being an intermediated messenger between DNA and proteins. As the appreciation for the importance of ncRNAs continues to emerge, it is also increasingly clear that these play critical roles in gene regulatory processes during development and differentiation. Further, regulatory RNAs are useful biomarkers for diagnosis of diseases. Hence these RNA regulators are essential to the development of therapeutics. This book on “Regulatory RNAs” offers a comprehensive view on our current understanding of these regulatory RNAs viz. siRNA, miRNA, piRNA, snoRNA, long non-coding RNA, small RNA etc. It addresses both the biogenesis and mechanism of action of regulatory RNAs with a primary focus on their annotation, experimental methodologies (microarray, next-gen sequencing etc.) for their discovery, computational tools for their prediction, and above all, applications of these revolutionary regulatory molecules in understanding biological systems and diseases, including therapeutics. This comprehensive volume is intended for readers with research or teaching interests in ncRNA biology and will provide a major information resource on current research in the fast-moving fields of RNA and gene expression regulation. Cutting-edge and concise, “Regulatory RNAs: Basics, Methods and Applications” promises to support vital research in the field of regulatory RNAs, ever-continuing to grow rapidly and gain increasing importance in basic and translational biology.

This volume contains 29 engrossing chapters contributed by worldwide, leading research groups in the field of chemical biology. Topics include pre-biology; the establishment of the genetic code; isomerization of RNA; damage of nucleobases in RNA; the dynamic structure of nucleic acids and their analogs in DNA replication, extra- and intra-cellular transport; molecular crowding by the use of ionic liquids; new technologies enabling the modification of gene expression via editing of therapeutic genes; the use of riboswitches; the modification of mRNA cap regions; new approaches to detect appropriately modified RNAs with EPR spectroscopy and the use of parallel and high-throughput techniques for the analysis of the structure and new functions of nucleic acids. This volume discusses how chemistry can add new frontiers to the field of nucleic acids in molecular medicine, biotechnology and nanotechnology and is not only an invaluable source of information to chemists, biochemists and life scientists but will also stimulate future research.