A wide range of new technology involving a direct genetic approach to the treatment of disease using nucleic acid therapeutics is the main focus of this absorbing book. Provides in-depth coverage of the rapid progress of this new technology, from its theoretical stage to its current position at the verge of experimental applications, along with provocative discussions of the therapeutic implications.

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.

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.

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 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.

With contributions from leading experts, this book is the first to focus solely on addressing the problems and reviewing the strategies currently being used to improve the delivery of antisense nucleic acids. Important delivery issues, such as improving biological stability, improving cell-specific targeting and cellular uptake, manipulating subcellular distribution and producing liposomal delivery systems for antisense agents are comprehensively covered in this volume. This book links review-type articles with contributions that contain exciting never-before-published data on the cellular delivery of oligonucleotides. It stimulates reading for both established researchers and newcomers to the antisense field.