Therapeutic Applications Of Rnai

The phenomenon of RNA interference has rapidly moved from groundbreaking scientific discovery to promising therapeutic approach. However, even as RNAi-based drugs enter the clinic, significant challenges remain, particulary in the area of delivery. Therapeutic Applications of RNAi provides detailed protocols in key areas of current focus, including testing of delivery vehicles, identification of appropriate model systems, and evaluation of the effects of RNAi in vivo. Produced by a team of internationally renowned authors, the volume describes the therapeutic applications of RNAi and potential pitfalls in oncology, viral infections and CNS disease, using a variety of delivery methods, including liposomes, peptide-based nanoparticles, polycationic polymers, and viral vehicles. Written in the highly successful Methods in Molecular BiologyTM series format, the chapters include brief introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and notes on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Therapeutic Applications of RNAi is an ideal guide for scientists attempting to solve the numerous challenges in this field and revolutionize the treatment of disease.

Commonly used by researchers to develop technologies for modifying and studying genetic process, RNA interference (RNAi) has many potential uses in medicine, biotechnology, and functional genomics. This book covers all essential aspects involved in the development of RNAi therapeutics, providing detailed guidance on the challenges and opportunities of bringing RNAi technologies from bench to clinic. It explores the design and mechanism of RNAi molecules, delivery strategies, and therapeutic applications in various diseases. Preclinical, regulatory, market, and intellectual aspects of RNAi technologies are also covered.

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.

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.

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

Since the discovery of RNA interference (RNAi) in 1998, research on the topic has advanced at an impressive pace. Small RNAs and, in particular, micro RNAs (miRNAs) play a fundamental role in gene regulation through the activation of RNAi. Detailed insights into the mechanisms of RNAi have led to an improved understanding of gene regulation in normal and disease states, and thereby enabled the exploitation of RNAi for a variety of applications. In this book, an international panel of RNAi experts critically reviews the most interesting advances in basic applied RNAi research, highlighting the applications in RNAi-based therapies and discussing the technical hurdles that remain. Topics covering the fundamental biological aspects of applied RNAi research include: the role of miRNAs in trinucleotide repeat disorders * miRNAs and HIV pathogenesis * miRNAs for epigenetic gene silencing * the role of miRNAs in virus-related cancers * non-canonical miRNA biogenesis. In the area of RNAi-based therapy, topics include: harnessing RNAi for the treatment of viral infections * optimizing the design of exogenous RNAi activators * blocking miRNA function with synthetic agents * somatic cell reprogramming * high-content miRNA-based screening tools * the use of miRNA target sites for control of transgene expression. The book will be essential reading for everyone involved in RNAi research, drug discovery and delivery, biomedical engineering, and biomaterials. Subject: Molecular Biology, Genomics, Life Science]

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

RNA interference (RNAi) is a genetic regulatory mechanism that occurs in eukaryotic cells and regulates the activity of genes. Its main function is to suppress, silence or deactivate genes. RNAi can occur either naturally or artificially. It occurs naturally through the production of pre-miRNA. RNAi can also be induced experimentally by using double-stranded RNA (dsRNA). The synthetic dsRNA may be of two types: small hairpin RNA (shRNA) or short interfering RNA (siRNA). An enzyme known as DICER is necessary in both natural and artificial pathways for the formation of miRNA from pre-miRNA or for the formation of siRNA from shRNA. This book traces the progress of this field and highlights some of its key concepts and applications. It studies, analyses and upholds the pillars of RNA interference and its utmost significance in modern times. This book includes contributions of experts and scientists which will provide innovative insights into this field.