Description: Rather than describing mechanisms of antisense action, this book focuses on current and future therapeutic uses of antisense oligonucleotides. It is organized into disease states, which shows the potential broad applications of these therapeutics to many disease states. This second edition updates the first edition published in 1996.
Purpose: The purpose is to address the potential use of antisense oligonucleotides as therapeutic agents to treat diseases in humans. A comprehensive review of current and past clinical trials is needed to show that these agents are more than just tools for research, but are potentially valuable therapeutic drugs. This book clearly demonstrates that the field of antisense research has significantly progressed in the past 10 years towards the goal of making antisense oligonucleotides valuable therapeutic drugs.
Audience: The book would be useful for both researchers and practitioners who have an interest in understanding the potential therapeutic utility of these compounds. The author is clearly an authority in the field and has extensive knowledge about the potential of antisense therapeutics.
Features: Antisense approaches that are currently under investigation to treat various diseases are covered. In particular, this book discusses the usefulness of antisense to treat cardiovascular disease, cancer, and various gastrointestinal disorders. What is useful to readers is that each chapter discusses the experimental approach that each investigator took to evaluate the usefulness of these agents to treat different disease states. Beyond this, the book also evaluates newer methods of antisense inhibition, such as ribozymes and siRNAs, and discusses the potential of these agents as therapeutics in the future. It might have been useful, however, to include one or two chapters on the use of siRNAs in vivo since this new mechanism of antisense action was discussed in the book; there are many such examples in the literature. One shortcoming is that many of the chapters begin with a recapitulation of the mechanism by which antisense oligonucleotides work. Since the first two chapters address this in detail, this could have been eliminated from each of the chapters.
Assessment: Antisense oligonucleotides are used by most investigators as tools for research. What is often lost is the potential of these compounds as drugs to treat diseases. This book does an excellent job of showing the reader that antisense oligonucleotides could be valuable therapeutic drugs in the future. The first edition focused primarily on mechanisms of antisense action. Therefore, this edition was needed to show how antisense research has progressed towards the clinic in the years since.