Antisense Therapy

Author : Nicolay Ferrari
Publisher : John Wiley & Sons
ISBN 13 : 1118537335
Total Pages : 576 pages
Book Rating : 4.36/5 ( download)

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Download or read book Oligonucleotide-Based Drugs and Therapeutics written by Nicolay Ferrari and published by John Wiley & Sons. This book was released on 2018-07-31 with total page 576 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 : Shashwat Sharad
Publisher : BoD – Books on Demand
ISBN 13 : 1789845327
Total Pages : 126 pages
Book Rating : 4.27/5 ( download)

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Download or read book Antisense Therapy written by Shashwat Sharad and published by BoD – Books on Demand. This book was released on 2019-11-20 with total page 126 pages. Available in PDF, EPUB and Kindle. Book excerpt: Antisense Therapy offers a comprehensive, state-of-the art perspective on the role of antisense therapy in the treatment of human disease, with a special focus on cancer. Use of antisense oligonucleotides is a growing field of pharmaceutical and biotech companies and research programs for treatment of several diseases. This book summarizes and presents the best updates, therapeutic principles, methods, and applications in the field and offers meaningful information to move treatment discovery forward.

Author : Stanley T. Crooke
Publisher : Springer Science & Business Media
ISBN 13 : 3642587852
Total Pages : 646 pages
Book Rating : 4.56/5 ( download)

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Download or read book Antisense Research and Application written by Stanley T. Crooke and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 646 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Author : Eric Wickstrom
Publisher : CRC Press
ISBN 13 : 1000145956
Total Pages : 452 pages
Book Rating : 4.53/5 ( download)

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Download or read book Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors written by Eric Wickstrom and published by CRC Press. This book was released on 2020-08-13 with total page 452 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Author : Clévio Nóbrega
Publisher : Springer
ISBN 13 : 3319717790
Total Pages : 469 pages
Book Rating : 4.91/5 ( download)

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Download or read book Polyglutamine Disorders written by Clévio Nóbrega and published by Springer. This book was released on 2018-02-09 with total page 469 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 13 : 1439832501
Total Pages : 848 pages
Book Rating : 4.09/5 ( download)

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Download or read book Antisense Drug Technology written by Stanley T. Crooke and published by CRC Press. This book was released on 2007-07-25 with total page 848 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 : Sudhir Agrawal
Publisher :
ISBN 13 : 9781592595853
Total Pages : 276 pages
Book Rating : 4.55/5 ( download)

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Download or read book Antisense Therapeutics written by Sudhir Agrawal and published by . This book was released on 1996 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: Annotation Dr. Agrawal's newest book is of significant importance since oligonucleotide-based drugs have the advantage of retaining their recognition for the target RNA, and provide the tools needed to modulate the expression of specific genes, making Antisense Therapeutics an essential resource for all antisense researchers.

Author : Margaret M. McCarthy
Publisher : Springer Science & Business Media
ISBN 13 : 1461549337
Total Pages : 179 pages
Book Rating : 4.38/5 ( download)

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Download or read book Modulating Gene Expression by Antisense Oligonucleotides to Understand Neural Functioning written by Margaret M. McCarthy and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 179 pages. Available in PDF, EPUB and Kindle. Book excerpt: When first introduced, antisense oligonucleotides were hailed as the long awaited magic bullet that would provide an unprecedented level of specificity in controlling gene expression. Following this initial enthusiasm, antisense oligonucleotides have been maligned as nonspecific, toxic, and essentially useless. However, application of antisense oligonucleotide technology in the nervous system stands apart from the use of this technique in peripheral systems, largely because of its enormous success. The source of this success remains a matter of some controversy. Modulating Gene Expression by Antisense Oligonucleotides to Understand Neural Functioning addresses the origins of that controversy and determines whether the nervous system is a privileged site for antisense oligonucleotide action and not subject to the same vagaries and pitfalls as non-neuronal systems. Modulating Gene Expression by Antisense Oligonucleotides to Understand Neural Functioning contains chapters by experts in the field that focus on the use of this technique in a variety of behavioral systems, as well as rapid and nonspecific effects and the uptake and metabolism of antisense oligonucleotides by the nervous system. Modulating Gene Expression by Antisense Oligonucleotides to Understand Neural Functioning features: experts in the field reporting on the use of antisense oligonucleotide technology in a variety of behavioral systems, including pain control, circadian rhythms, ingestion and control of water balance, and reproductive behaviors; the novel use of plasmids to express antisense RNA in the nervous system; the biodistribution and metabolism of antisense oligonucleotides in the nervous system; rapid and unusual effects; and non-specific effects.

Author : Jens Kurreck
Publisher : Royal Society of Chemistry
ISBN 13 : 0854041168
Total Pages : 362 pages
Book Rating : 4.69/5 ( download)

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Download or read book Therapeutic Oligonucleotides written by Jens Kurreck and published by Royal Society of Chemistry. This book was released on 2008 with total page 362 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a compelling overall update on current status of RNA interference

Author : Saghir Akhtar
Publisher : CRC Press
ISBN 13 : 1351366130
Total Pages : 393 pages
Book Rating : 4.37/5 ( download)

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Download or read book Delivery Strategies for Antisense Oligonucleotide Therapeutics written by Saghir Akhtar and published by CRC Press. This book was released on 2017-07-28 with total page 393 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.