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Author: Olof Gissberg
Publisher: Humana
ISBN: 9781493996728
Category : Medical
Languages : en
Pages : 344
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Book Description
This book provides a collection of comprehensive, up-to-date, and broadly applicable guides to the research and development fields of oligonucleotide (ON) therapeutics. Covering topics from the study of antisense and anti-gene effects to oligonucleotides in the context of drug discovery and development, the volume explores a wide-ranging and useful spectrum of methods and protocols needed to take full advantage of therapeutic applications involving ONs. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Oligonucleotide-Based Therapies: Methods and Protocols aims to be a great aid in the laboratory as well as an ideal reference guide when designing antisense and anti-gene oligonucleotides for therapeutic applications.
Author: Olof Gissberg
Publisher: Humana
ISBN: 9781493996728
Category : Medical
Languages : en
Pages : 344
Get Book Here
Book Description
This book provides a collection of comprehensive, up-to-date, and broadly applicable guides to the research and development fields of oligonucleotide (ON) therapeutics. Covering topics from the study of antisense and anti-gene effects to oligonucleotides in the context of drug discovery and development, the volume explores a wide-ranging and useful spectrum of methods and protocols needed to take full advantage of therapeutic applications involving ONs. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Oligonucleotide-Based Therapies: Methods and Protocols aims to be a great aid in the laboratory as well as an ideal reference guide when designing antisense and anti-gene oligonucleotides for therapeutic applications.
Author: Nicolay Ferrari
Publisher: John Wiley & Sons
ISBN: 1118537335
Category : Medical
Languages : en
Pages : 576
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Book Description
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.
Author: Jens Kurreck
Publisher: Royal Society of Chemistry
ISBN: 0854041168
Category : Medical
Languages : en
Pages : 362
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Book Description
This book provides a compelling overall update on current status of RNA interference
Author: Clévio Nóbrega
Publisher: Springer
ISBN: 3319717790
Category : Medical
Languages : en
Pages : 467
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Book Description
This book provides a cutting-edge review of polyglutamine disorders. It primarily focuses on two main aspects: (1) the mechanisms underlying the pathologies’ development and progression, and (2) the therapeutic strategies that are currently being explored to stop or delay disease progression. Polyglutamine (polyQ) disorders are a group of inherited neurodegenerative diseases with a fatal outcome that are caused by an abnormal expansion of a coding trinucleotide repeat (CAG), which is then translated in an abnormal protein with an elongated glutamine tract (Q). To date, nine polyQ disorders have been identified and described: dentatorubral-pallidoluysian atrophy (DRPLA); Huntington’s disease (HD); spinal–bulbar muscular atrophy (SBMA); and six spinocerebellar ataxias (SCA 1, 2, 3, 6, 7, and 17). The genetic basis of polyQ disorders is well established and described, and despite important advances that have opened up the possibility of generating genetic models of the disease, the mechanisms that cause neuronal degeneration are still largely unknown and there is currently no treatment available for these disorders. Further, it is believed that the different polyQ may share some mechanisms and pathways contributing to neurodegeneration and disease progression.
Author: Stanley T. Crooke
Publisher: CRC Press
ISBN: 1439832501
Category : Medical
Languages : en
Pages : 858
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Book Description
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
Author: Marcelo Larramendy
Publisher: BoD – Books on Demand
ISBN: 9535122649
Category : Science
Languages : en
Pages : 222
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Book Description
This edited book, "Nucleic Acids - From Basic Aspects to Laboratory Tools", contains a series of chapters that highlight the development and status of the various aspects of the nucleic acids related to DNA chemistry and biology and the molecular application of these small DNA molecules and related synthetic analogues within biological systems. Furthermore, it is hoped that the information in the present book will be of value to those directly engaged in the handling and use of nucleic acids, and that this book will continue to meet the expectations and needs of all who are interested in the different fascinating aspects of molecular biology.
Author: Keiko Hiyama
Publisher: Springer Science & Business Media
ISBN: 1603278796
Category : Medical
Languages : en
Pages : 375
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Book Description
Telomerase, an enzyme that maintains telomeres and endows eukaryotic cells with immortality, was first discovered in tetrahymena in 1985. In 1990s, it was proven that this enzyme also plays a key role in the infinite proliferation of human cancer cells. Now telomere and telomerase are widely accepted as important factors involved in cancer biology, and as promising diagnostic tools and therapeutic targets. Recently, role of telomerase in “cancer stem cells” has become another attractive story. Until now, there are several good books on telomere and telomerase focusing on biology in ciliates, yeasts, and mouse or basic sciences in human, providing basic scientists or students with updated knowledge.
Author: Joy A. Cavagnaro
Publisher: CRC Press
ISBN: 1000471853
Category : Medical
Languages : en
Pages : 952
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Book Description
Translational Medicine: Optimizing Preclinical Safety Evaluation of Biopharmaceuticals provides scientists responsible for the translation of novel biopharmaceuticals into clinical trials with a better understanding of how to navigate the obstacles that keep innovative medical research discoveries from becoming new therapies or even making it to clinical trials. The book includes sections on protein-based therapeutics, modified proteins, oligonucleotide-based therapies, monoclonal antibodies, antibody–drug conjugates, gene and cell-based therapies, gene-modified cell-based therapies, combination products, and therapeutic vaccines. Best practices are defined for efficient discovery research to facilitate a science-based, efficient, and predictive preclinical development program to ensure clinical efficacy and safety. Key Features: Defines best practices for leveraging of discovery research to facilitate a development program Includes general principles, animal models, biomarkers, preclinical toxicology testing paradigms, and practical applications Discusses rare diseases Discusses "What-Why-When-How" highlighting different considerations based upon product attributes. Includes special considerations for rare diseases About the Editors Joy A. Cavagnaro is an internationally recognized expert in preclinical development and regulatory strategy with an emphasis on genetic medicines.. Her 40-year career spans academia, government (FDA), and the CRO and biotech industries. She was awarded the 2019 Arnold J Lehman Award from the Society of Toxicology for introducing the concept of science-based, case-by-case approach to preclinical safety evaluation, which became the foundation of ICH S6. She currently serves on scientific advisory boards for advocacy groups and companies and consults and lectures in the area of preclinical development of novel therapies. Mary Ellen Cosenza is a regulatory toxicology consultant with over 30 years of senior leadership experience in the biopharmaceutical industry in the U.S., Europe, and emerging markets. She has held leadership position in both the American College of Toxicology (ACT) and the International Union of Toxicology (IUTOX) and is also an adjunct assistant professor at the University of Southern California where she teaches graduate-level courses in toxicology and regulation of biologics.
Author: Arnold von Eckardstein
Publisher: Springer
ISBN: 9783319096643
Category : Medical
Languages : en
Pages : 0
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Book Description
In this Handbook of Experimental Pharmacology on “High Density Lipoproteins – from biological understanding to clinical exploitation” contributing authors (members of COST Action BM0904/HDLnet) summarize in more than 20 chapters our current knowledge on the structure, function, metabolism and regulation of HDL in health and several diseases as well as the status of past and ongoing attempts of therapeutic exploitation. The book is of interest to researchers in academia and industry focusing on lipoprotein metabolism, cardiovascular diseases and immunology as well as clinical pharmacologists, cardiologists, diabetologists, nephrologists and other clinicians interested in metabolic or inflammatory diseases.
Author: Sudhir Agrawal
Publisher: Royal Society of Chemistry
ISBN: 1788017323
Category : Science
Languages : en
Pages : 566
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Book Description
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.
