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.

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.

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.

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

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.

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