Antisense-Based Drugs and Therapeutics: Preclinical and Clinical Considerations for Development

This book comprehensively reviews up-to-date principles, methods, and applications of antisense-based drugs and therapeutics. It provides fundamental knowledge about the toxicology and chemistry of antisense oligonucleotides, an increasingly popular area in the field of drugs and therapeutics. The book is divided into 6 parts that cover the basics, toxicological properties, general ADME characteristics, preclinical toxicology, clinical safety assessment, and other classes of oligonucleotides. The book starts with an introduction and a foreword that covers the evolution of antisense oligonucleotides, from concept to development and the importance of preclinical studies and safety assessment. Beginning chapters highlight mechanisms, medicinal chemistry, pharmacological properties, administration and formulations of delivery, regulatory perspectives, and various types of toxicological effects. Following chapters provide in-depth detail on innovative topics and their relation to antisense therapeutics, like pharmacokinetics and pharmacodynamics, toxicokinetics, genotoxicity, species selection, exaggerated pharmacology, reproductive toxicity and carcinogenicity, vaccine adjuvents, siRNA, splice-switching oligonucleotides, and aptamers. Overall, 20 chapters of detailed discussion review and connect recent developments in the field and provide room for further research and advancement.