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Oligonucleotide–Based Drugs and Therapeutics – Preclinical and Clinical Considerations for Development

Autor N Ferrari
en Limba Engleză Hardback – 6 sep 2018
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.
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Specificații

ISBN-13: 9781118537336
ISBN-10: 1118537335
Pagini: 576
Dimensiuni: 152 x 229 x 32 mm
Greutate: 0.9 kg
Editura: Wiley
Locul publicării:Hoboken, United States

Public țintă

Scientists in the pharmaceutical and biotechnology industry, such as chemists, pharmaceutical scientists, and toxicologists
Medicinal chemists, biochemists, molecular chemists, nucleic acid chemists, chemical biologists
Pharmaceutical researchers, companies, and laboratories
Regulatory specialists and safety assessment professionals
Industry and academic professionals and advanced graduate students interested in novel therapeutics, antisense therapy, and oligonucleotides

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Descriere

Antisense-based drugs are part of a growing number of pharmaceutical and biotech programs to treat diseases including cancer, diabetes, and ALS. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide from the safety assessment to clinical experience with antisense oligonucleotides.