For Stanford scientists, RNAi gene therapy takes two steps forward, one step back

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.

RNA interference (RNAi), in which RNA silences RNA, is the most recent discovery to revolutionize the study of biology. In RNAi: Design and Applications, leaders in the field contribute state-of-the-art, easy to follow methods and bench protocols designed for practical, everyday use of RNAi in biological research. Divided into two parts, this comprehensive volume covers fundamentals including designs of RNAi, biochemical assay protocols for the major components of RNAi, and study of potential off-target effects, followed by an extensive section covering various applications of RNAi in diverse model organisms and systems, from antiviral and anticancer applications to altering flower color in plants.

Cutting edge and clearly written, RNAi: Design and Applications enables a...

One of the major recent discoveries in molecular and cellular biology is that small double-stranded RNA molecules selectively turn off gene expression in all types of cell, a phenomenon known as RNA silencing. This discovery led to the development of RNA interference (known as RNAi) as a powerful research tool in the functional study of individual genes and their products, and in functional genomics. In RNAi, specific small double-stranded RNA molecules (small interfering RNAs or siRNAs) are introduced into cells to selectively silence certain genes.

RNAi covers the basic concepts and mechanisms of RNAi, transfection of cells with siRNAs, the design and validation of RNAi reagents, RNAi techniques in different organisms, large-scale RNAi screening, applications of RNAi in...

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...

Chapters of the book discuss RNAi in plants, Drosophila, C. elegans, and mammalian systems. As other "Nuts&Bolts series" books, RNA Intereference offeres detailed protocols and experimental tips.