Applied Antisense Oligonucleotide Technology PDF Download

Author: Cy A. Stein
Publisher: Wiley-Liss
ISBN:
Category : Medical
Languages : en
Pages : 556

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Book Description
Applied Antisense Oligonucleotide Technology provides the basic concepts as well as the practical concerns associated with the use of antisense oligonucleotides to modify gene expression. Chapters include oligonucleotide chemistry, DNA triplex formation, delivery mechanisms, pharmocokinetics, toxicity, oligonucleotides.

Author: Stanley T. Crooke
Publisher: CRC Press
ISBN: 1439832501
Category : Medical
Languages : en
Pages : 848

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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: Stanley T. Crooke
Publisher: Springer Science & Business Media
ISBN: 3642587852
Category : Medical
Languages : en
Pages : 646

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Book Description
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: 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: Stanley T. Crooke
Publisher: CRC Press
ISBN: 9780849347054
Category : Medical
Languages : en
Pages : 620

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Book Description
Antisense Research and Applications is a comprehensive review of oligonucleotide research covering molecular biological advances in this field, the current status of antisense drug research, and strategies for future research and therapeutic applications. In bringing together the latest research from an array of authoritative scientists, Antisense Research and Applications provides an integrated conceptual basis for considering oligonucleotide therapeutics. Topics covered in the 32 chapters of this book include nucleic acid structure and function, antisense RNA, medicinal chemistry of oligonucleotides, analogs, pharmacokinetics and toxicology, and activities of current antisense drugs. This volume addresses advances in a broad range of disciplines and is an excellent resource for basic researchers and applied investigators in pharmaceutical laboratories and in such fields as biochemistry and molecular biology.

Author:
Publisher: Elsevier
ISBN: 0080496717
Category : Science
Languages : en
Pages : 683

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Book Description
Antisense technology is the ability to manipulate gene expression within mammalian cells providing powerful experimental approaches for the study of gene function and gene regulation. For example, methods which inhibit gene expression permit studies probing the normal function of a specific product within a cell. Such methodology can be used in many disciplines such as pharmacology, oncology, genetics, cell biology, developmental biology, molecular biology, biochemistry, and neurosciences. This volume will be a truly important tool in biomedically-oriented research. The critically acclaimed laboratory standard for more than forty years, Methods in Enzymology is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. Now with more than 300 volumes (all of them still in print), the series contains much material still relevant today-truly an essential publication for researchers in all fields of life sciences.

Author: Conrad Lichtenstein
Publisher: IRL Press
ISBN: 9780199635832
Category : Antisense DNA.
Languages : en
Pages : 334

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Book Description
Antisense technology is a powerful procedure that permits the controlled silencing of a specific gene for investigations of mRNA and protein function. This valuable text provides proven step-by-step protocols for antisense techniques in a range of different organisms and cell culture systems. In addition it discusses the potential benefits and problems for various antisense methods which complement gene knock-out experiments. The book includes antisense techniques such as: analysis of nucleic acid structures; measurement and evaluation of antisense effects; selection, preparation and the use of antisense oligonucleotides; in vitro RNA transcription; construction strategies, testing and optimization of catalytic antisense RNAs based on hammerhead ribozymes; synthesis and evaluation of 2-5 A- antisense chimeras for targeted degradation of RNA. The application of these technologies are then described, with chapters on antisense techniques in IDictyostelium, plants and the medical uses and benefits of antisense sense technology both in vitro and in vivo. Finally, non-antisense effects of oligonucleotides, "anti-sense rescue", shotgun antisense mutagenesis, and in vivo, in vitro and in situ detection of sense: antisense duplexes by structure-specific anti-RNA antibodies are discussed. The numerous hints and tips for success and advice on trouble shooting, make this authoritative text invaluable to all those researchers who work on gene expression, RNA transcription, or protein function.

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: R. Schlingensiepen
Publisher: Wiley-Blackwell
ISBN: 9780865426696
Category : Medical
Languages : en
Pages : 365

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Book Description
The target specific inhibition of gene-expression using antisense nucleic acids has developed into a key technology in molecular biology, bio-medical research and an entirely new field of pharmacotherapy. Like the first conference in 1993, the Second International Conference on Antisense Nucleic Acids will provide a forum for basic researchers, clinicians focusing on the application of antisense techniques in physiological settings and disease states. Major topics are Drug Delivery Systems, Pharmacology Targeting of sites of pathology and reports on the first clinical trials in virology and hematology.

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.