The sequencing of the human genome and subsequent elucidation of the molecular pathways that are important in the pathology of disease have provided unprecedented opportunities for the development of new therapeutics. Nucleic acid-based drugs have emerged in recent years to yield extremely promising candidates for drug therapy to a wide range of diseases. Advances in Nucleic Acid Therapeutics is a comprehensive review of the latest advances in the field, covering the background of the development of nucleic acids for therapeutic purposes to the array of drug development approaches currently being pursued using antisense, RNAi, aptamer, immune modulatory and other synthetic oligonucleotides. Nucleic acid therapeutics is a field that has been continually innovating to meet the challenges of drug discovery and development; bringing contributions together from leaders at the forefront of progress, this book depicts the many approaches currently being pursued in both academia and industry. A go-to volume for medicinal chemists, Advances in Nucleic Acid Therapeutics provides a broad overview of techniques of contemporary interest in drug discovery.
Table of Content
History and Development of Nucleotide Analogues in Nucleic Acids Drugs; Mechanisms of Antisense Oligonucleotides; The Medicinal Chemistry of RNase H-activating Antisense Oligonucleotides; Antisense Technology: Liver Targeting and Beyond for Drug Discovery; Oligonucleotide-based Toll-like Receptor Antagonists and Therapeutic Applications; Splicing Modulation for Therapeutics; Targeting Toxic Repeats; Research and Development of Oligonucleotides Targeting Micro RNAs (mi RNAs); Oligonucleotide Targeting of Long Non-coding RNAs; Conjugate-mediated Delivery of RNAi-based Therapeutics: Enhancing Pharmacokinetics-Pharmacodynamics Relationships of Medicinal Oligonucleotides; Liver-targeted RNAi Therapeutics: Principles and Applications; Advances and Challenges of RNAi-Based Anti-HIV Therapeutics; Nucleic Acid Innate Immune Receptors; Synthetic Agonists of Toll-like Receptors and Therapeutic Applications; Prostate-specific Membrane Antigen (PSMA) Aptamers for Prostate Cancer Imaging and Therapy; Aptamers and Clinical Applications; CRISPR-based Technologies for Genome Engineering: Properties, Current Improvements and Applications in Medicine; Therapeutic Potential of Ribozymes; Large-scale Automated Synthesis of Therapeutic Oligonucleotides: A Status Update; Preclinical and Clinical Drug-metabolism, Pharmacokinetics and Safety of Therapeutic Oligonucleotides; Subject Index
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