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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.

MicroRNAs (miRNAs), endogenous noncoding regulatory mRNAs of around 22-nucleotides long, have rapidly emerged as one of the key governors of the gene expression regulatory program in cells of varying species, with ever-increasing implications in the control of the fundamental biological processes and in the pathogenesis of adult humans. The exciting findings in this field have inspired us with a premise and a promise that miRNAs will ultimately be taken to the heart for therapy of human disease. While miRNAs have been considered potential therapeutic targets for disease treatment, it remains obscured what strategies we can use to achieve the goal. In the past years, we have witnessed a rapid evolving of many creative, innovative, inventive strategies and methodologies pertinent to miRNA research and applications. These technologies have convincingly demonstrated their efficacy and reliability in producing gain-of-function or loss-of-function of miRNAs through targeting miRNA expression/biogenesis/function, providing new tools for elucidating miRNA functions and opening up a new avenue for the development of new agents targeting miRNAs for therapeutic aims. The present book provides comprehensive descriptions of these technologies and their applications to miRNA research and to new drug design for miRNA-related diseases. It starts with an overview of up-to-date knowledge of miRNA biology and the potential of miRNAs as therapeutic targets for human disease, followed by an introduction of the new concept of miRNA interference (miRNAi) and the perspectives of miRNAi technologies in general terms. In the following, each chapter introduces one of the miRNAi technologies with detailed descriptions of state-of-the-art design, procedures, principles and applications to basic research, R and D and clinical therapy.

In the last few years the major effect that RNAi has had in invertebrate systems like C.elegans and drosophila is beginning to take hold in mammalian systems through both single gene knockdown experiments and genome-scale screens. In the next decade, there will no doubt be both notable successes and failures as we attempt to apply this genetic tool to various biological problems for the first time in academia and industry. Through the introduction of RNAi, mammalian systems have finally gained admittance to the pantheon of model genetic systems.