Antisense Research and Applications is a comprehensive review of oligonucleotide research covering molecular biological advances in this field, the current status of antisense drug research, and strategies for future research and therapeutic applications. In bringing together the latest research from an array of authoritative scientists, Antisense Research and Applications provides an integrated conceptual basis for considering oligonucleotide therapeutics. Topics covered in the 32 chapters of this book include nucleic acid structure and function, antisense RNA, medicinal chemistry of oligonucleotides, analogs, pharmacokinetics and toxicology, and activities of current antisense drugs. This volume addresses advances in a broad range of disciplines and is an excellent resource for basic researchers and applied investigators in pharmaceutical laboratories and in such fields as biochemistry and molecular biology.

Antisense technology may result in dramatic changes in the therapy of many diseases and may provide tools to dissect pharmacological processes and to confirm the roles of various genes. In this volume, progress in the understanding of antisense technology and its use in creating new drugs is discussed. Potential caveats, pitfalls and limitations of the technology are also presented. In the next few years the pace at which new molecular targets will be identified will increase exponentially as the sequencing of the human genome and of other genomes proceeds.

Extensively revised and updated, Antisense Drug Technology: Principles, Strategies, and Applications, Second Edition reflects the logarithmic progress made in the past four years of oligonucleotide-based therapies, and, in particular, antisense therapeutics and research. Interpreting lessons learned from the clinical trials of first generati

A comprehensive review of contemporary antisense oligonucleotides drugs and therapeutic principles, methods, applications, and research Oligonucleotide-based drugs, in particular antisense oligonucleotides, are part of a growing number of pharmaceutical and biotech programs progressing to treat a wide range of indications including cancer, cardiovascular, neurodegenerative, neuromuscular, and respiratory diseases, as well as other severe and rare diseases. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide covering all key aspects of this increasingly popular area of pharmacology and biotech and pharma research, from the basic science behind antisense oligonucleotides chemistry, toxicology, manufacturing, to safety assessments, the design of therapeutic protocols, to clinical experience. Antisense oligonucleotides are single strands of DNA or RNA that are complementary to a chosen sequence. While the idea of antisense oligonucleotides to target single genes dates back to the 1970's, most advances have taken place in recent years. The increasing number of antisense oligonucleotide programs in clinical development is a testament to the progress and understanding of pharmacologic, pharmacokinetic, and toxicologic properties as well as improvement in the delivery of oligonucleotides. This valuable book reviews the fundamentals of oligonucleotides, with a focus on antisense oligonucleotide drugs, and reports on the latest research underway worldwide. • Helps readers understand antisense molecules and their targets, biochemistry, and toxicity mechanisms, roles in disease, and applications for safety and therapeutics • Examines the principles, practices, and tools for scientists in both pre-clinical and clinical settings and how to apply them to antisense oligonucleotides • Provides guidelines for scientists in drug design and discovery to help improve efficiency, assessment, and the success of drug candidates • Includes interdisciplinary perspectives, from academia, industry, regulatory and from the fields of pharmacology, toxicology, biology, and medicinal chemistry Oligonucleotide-Based Drugs and Therapeutics belongs on the reference shelves of chemists, pharmaceutical scientists, chemical biologists, toxicologists and other scientists working in the pharmaceutical and biotechnology industries. It will also be a valuable resource for regulatory specialists and safety assessment professionals and an important reference for academic researchers and post-graduates interested in therapeutics, antisense therapy, and oligonucleotides.

In the past few years, antisense methodology has moved from in vitro studies to in vivo studies and first human trials. While the basic concept of antisense technology is simple, the methodological problems associated with its use are numerous and complex. Antisense- based methods have proven to be a field of research where careful attention to experimental protocols and appropriate controls is necessary. The Manual of Antisense Methodology emphasizes the application of antisense oligonucleotides, and is a guide for the identification of antisense and non-antisense effects in different experimental settings. The work is organized into three sections: antisense application in vitro, antisense application in vivo (animal models) and finally, clinical antisense studies. Where at all possible, the methods are described in sufficient detail to allow reproduction of a given experiment. The Manual of Antisense Methodology will be of interest to researchers in immunology, cancer research, pharmacology and internal medicine; and physicians conducting clinical studies in these fields.

Antisense technology is the ability to manipulate gene expression within mammalian cells providing powerful experimental approaches for the study of gene function and gene regulation. For example, methods which inhibit gene expression permit studies probing the normal function of a specific product within a cell. Such methodology can be used in many disciplines such as pharmacology, oncology, genetics, cell biology, developmental biology, molecular biology, biochemistry, and neurosciences. This volume will be a truly important tool in biomedically-oriented research.The critically acclaimed laboratory standard for more than forty years, Methods in Enzymology is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. Now with more than 300 volumes (all of them still in print), the series contains much material still relevant today-truly an essential publication for researchers in all fields of life sciences.

Applied Antisense Oligonucleotide Technology provides the basic concepts as well as the practical concerns associated with the use of antisense oligonucleotides to modify gene expression. Chapters include oligonucleotide chemistry, DNA triplex formation, delivery mechanisms, pharmocokinetics, toxicity, oligonucleotides.

This volume is unique to the existing literature in the Peptide Nucleic Acid field, in that it focuses on comparing and contrasting PNA with other available oligonucleotide homologues and considers areas in which these biomolecules could be profitably applied to clinical and diagnostic applications. Part I of the book addresses comparative strengths and weaknesses of various nucleoside homologues. Part II of the book addresses specific translational or clinical applications for PNA and related antisense biomolecules. The editors have succeeded in presenting a balanced yet broad view of the methods available for gene targeting and modification.

Antisense Therapy offers a comprehensive, state-of-the art perspective on the role of antisense therapy in the treatment of human disease, with a special focus on cancer. Use of antisense oligonucleotides is a growing field of pharmaceutical and biotech companies and research programs for treatment of several diseases. This book summarizes and presents the best updates, therapeutic principles, methods, and applications in the field and offers meaningful information to move treatment discovery forward.

A compilation of papers from many different scientific meetings in recent years, this reference source reviews the use of antisense nucleic acids to modulate or down-regulate gene expression, provides clues on the action mechanism of sense and antisense genes as well as data on antisense nucleic aci