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.

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.

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

The target specific inhibition of gene-expression using antisense nucleic acids has developed into a key technology in molecular biology, bio-medical research and an entirely new field of pharmacotherapy. Like the first conference in 1993, the Second International Conference on Antisense Nucleic Acids will provide a forum for basic researchers, clinicians focusing on the application of antisense techniques in physiological settings and disease states. Major topics are Drug Delivery Systems, Pharmacology Targeting of sites of pathology and reports on the first clinical trials in virology and hematology.

An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors. Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors furnishes a historical overview of genetic therapy highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications illustrates for new practitioners how each stage of genetic therapy is developed details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy and addresses questions of preparation, delivery, toxicity, mechanism, and specificity.

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.

Annotation Dr. Agrawal's newest book is of significant importance since oligonucleotide-based drugs have the advantage of retaining their recognition for the target RNA, and provide the tools needed to modulate the expression of specific genes, making Antisense Therapeutics an essential resource for all antisense researchers.

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 special report on Gene Therapy Companies by K.K. Jain provides an up-to-date overview of the gene therapy industry. It contains a wealth of very practical information on 105 companies. For each company, information is available on the people to contact, their contact details, a corporate history, the company's technologies and products, financial data, patents held, products in clinical trials, collaborations with other companies and selected publications from the company's scientists. Almost 200 companies were initially considered for inclusion but just over 100 companies were finally selected as they fulfilled the necessary criteria to be classified as a 'Gene Therapy Company,', defined broadly to include cell therapy companies. Not all of the companies devote their activity exclusively to gene therapy but they have sufficient relevance to be included. As part of this project, new companies were discovered that have not been reported in other publications or on the internet. Dr. Jain has spared no effort to ensure that the information is up-to-date and accurate at the time of publishing. In most instances, the information was reviewed by the companies to correct any outdated information or to add any complementary or recent information. In addition to the detailed descriptions of each company, the data is also presented in summary tabular form so that at a glance the reader can identify collaborations within the gene therapy industry, or determine which companies are pursuing strategies involving viral, non-viral or other approaches to gene therapy, and which diseases are being targetted. Given that much of the gene therapy research and development is currently being carried out within the industrial sector, it is important for scientists, managers and investors to have the relevant information at their fingertips. This special report provides that information in one accurate, easy to consult and up-to-date source.