Oligonucleotide Therapeutics

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Oligonucleotide-Based Drugs and Therapeutics

Author : Nicolay Ferrari
Publisher : John Wiley & Sons
ISBN 13 : 1118537335
Total Pages : 576 pages
Book Rating : 4.1/5 (185 download)

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Book Synopsis Oligonucleotide-Based Drugs and Therapeutics by : Nicolay Ferrari

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.

Oligonucleotide-Based Therapies

Author : Olof Gissberg
Publisher : Humana
ISBN 13 : 9781493996728
Total Pages : 344 pages
Book Rating : 4.9/5 (967 download)

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Book Synopsis Oligonucleotide-Based Therapies by : Olof Gissberg

Download or read book Oligonucleotide-Based Therapies written by Olof Gissberg and published by Humana. This book was released on 2020-08-15 with total page 344 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Synthesis of Therapeutic Oligonucleotides

Author : Satoshi Obika
Publisher : Springer
ISBN 13 : 981131912X
Total Pages : 284 pages
Book Rating : 4.8/5 (113 download)

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Book Synopsis Synthesis of Therapeutic Oligonucleotides by : Satoshi Obika

Download or read book Synthesis of Therapeutic Oligonucleotides written by Satoshi Obika and published by Springer. This book was released on 2018-12-08 with total page 284 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents the latest knowledge on a broad range of topics relating to the synthesis of natural and artificial oligonucleotides with therapeutic potential. Nucleic acid-based therapeutics are attracting much attention, and numerous therapeutic oligonucleotides, such as antisense oligonucleotides, siRNAs, splice-switching oligonucleotides, and nucleic acid aptamers, are being evaluated in clinical trials for the treatment of a variety of diseases. Synthesis of Therapeutic Oligonucleotides covers a broad range of topics in the field that are of high relevance to researchers, including the synthesis of natural and chemically modified oligonucleotides, the development of novel nucleic acid analogs, industrial scale synthesis and purification of oligonucleotides, and important aspects of chemistry, manufacturing, and controls (CMC). The aim is to provide new insights and inspire fresh ideas in nucleic acid chemistry that may ultimately lead to novel concepts and techniques and the discovery of more effective nucleic acid drugs. The book will be of high value for both established researchers in the field and students intending to specialize in nucleic acid chemistry research.

Delivery Strategies for Antisense Oligonucleotide Therapeutics

Author : Saghir Akhtar
Publisher : CRC Press
ISBN 13 : 1351366130
Total Pages : 393 pages
Book Rating : 4.3/5 (513 download)

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Book Synopsis Delivery Strategies for Antisense Oligonucleotide Therapeutics by : Saghir Akhtar

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.

Oligonucleotide-Based Drugs and Therapeutics

Author : Nicolay Ferrari
Publisher : John Wiley & Sons
ISBN 13 : 1119070295
Total Pages : 576 pages
Book Rating : 4.1/5 (19 download)

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Book Synopsis Oligonucleotide-Based Drugs and Therapeutics by : Nicolay Ferrari

Download or read book Oligonucleotide-Based Drugs and Therapeutics written by Nicolay Ferrari and published by John Wiley & Sons. This book was released on 2018-06-06 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.

Oligonucleotide Therapeutics

Author : Thomas Tuschl
Publisher : John Wiley & Sons
ISBN 13 : 1573317586
Total Pages : 101 pages
Book Rating : 4.5/5 (733 download)

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Book Synopsis Oligonucleotide Therapeutics by : Thomas Tuschl

Download or read book Oligonucleotide Therapeutics written by Thomas Tuschl and published by John Wiley & Sons. This book was released on 2009-10-19 with total page 101 pages. Available in PDF, EPUB and Kindle. Book excerpt: Oligonucleotides modulate gene-specific expression within cells and can be used to identify genes involved in diseases. Continuing developments in oligonucleotide research have begun to unleash their potential as therapeutic agents. Contributions from basic researchers in molecular biology, cell biology, nucleic acid chemistry, pharmacology, and applied therapeutics present new technologies in the field of oligonucleotide research and are organized into sections focusing on small RNA-mediated gene silencing, miRNA, oligonucleotide chemistry and new technologies, delivery strategies, immunorecognition of nucleic acids, drug and therapeutic development, aptamers and other evolved systems, and preclinical models and clinical programs. NOTE: Annals volumes are available for sale as individual books or as a journal. For information on institutional journal subscriptions, please visit www.blackwellpublishing.com/nyas. ACADEMY MEMBERS: Please contact the New York Academy of Sciences directly to place your order (www.nyas.org). Members of the New York Academy of Science receive full-text access to the Annals online and discounts on print volumes. Please visit http://www.nyas.org/MemberCenter/Join.aspx for more information about becoming a member.

Polyglutamine Disorders

Author : Clévio Nóbrega
Publisher : Springer
ISBN 13 : 3319717790
Total Pages : 469 pages
Book Rating : 4.3/5 (197 download)

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Book Synopsis Polyglutamine Disorders by : Clévio Nóbrega

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.

Therapeutic Oligonucleotides

Author : Jens Kurreck
Publisher : Royal Society of Chemistry
ISBN 13 : 0854041168
Total Pages : 362 pages
Book Rating : 4.8/5 (54 download)

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Book Synopsis Therapeutic Oligonucleotides by : Jens Kurreck

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

Oligonucleotides as Therapeutic Agents

Author : Derek J. Chadwick
Publisher : John Wiley & Sons
ISBN 13 : 0470515406
Total Pages : 260 pages
Book Rating : 4.4/5 (75 download)

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Book Synopsis Oligonucleotides as Therapeutic Agents by : Derek J. Chadwick

Download or read book Oligonucleotides as Therapeutic Agents written by Derek J. Chadwick and published by John Wiley & Sons. This book was released on 2008-04-30 with total page 260 pages. Available in PDF, EPUB and Kindle. Book excerpt: The use of oligonucleotides as therapeutic agents rests upon their ability to interfere, in a sequence-specific manner, with the fundamental machinery of protein synthesis either by binding to the mRNAs transcribed from a gene or by binding directly to a target gene. This approach can be used not only for inhibition of the synthesis of host proteins but also of those required by invading pathogens. Potential therapeutic applications are enormous, ranging over hypertension, cardiovascular disease, autoimmune disease, vital and other parasitic infections (especially HIV), and cancer. This book discusses the chemistry and pharmacokinetics of oligonucleotides and their analogues, and surveys the results of structure-activity studies and current clinical trials. It also critically reviews the problems with antisense therapy, such as the enzymatic destruction of oligonucleotides, the doses required for a therapeutic response, the difficulty in directing oligonucleotides to particular target tissues and cells, the need for parenteral administration, and doubts concerning the mechanism of action (especially problems associated with non-specific binding to proteins) and long-term effects.

Applied Antisense Oligonucleotide Technology

Author : Cy A. Stein
Publisher : Wiley-Liss
ISBN 13 :
Total Pages : 556 pages
Book Rating : 4.3/5 (91 download)

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Book Synopsis Applied Antisense Oligonucleotide Technology by : Cy A. Stein

Download or read book Applied Antisense Oligonucleotide Technology written by Cy A. Stein and published by Wiley-Liss. This book was released on 1998-03-19 with total page 556 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Advances in Nucleic Acid Therapeutics

Author : Michael J Gait
Publisher : Royal Society of Chemistry
ISBN 13 : 1788012097
Total Pages : 566 pages
Book Rating : 4.7/5 (88 download)

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Book Synopsis Advances in Nucleic Acid Therapeutics by : Michael J Gait

Download or read book Advances in Nucleic Acid Therapeutics written by Michael J Gait and published by Royal Society of Chemistry. This book was released on 2019-02-11 with total page 566 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Antisense Drug Technology

Author : Stanley T. Crooke
Publisher : CRC Press
ISBN 13 : 1439832501
Total Pages : 848 pages
Book Rating : 4.4/5 (398 download)

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Book Synopsis Antisense Drug Technology by : Stanley T. Crooke

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

Handbook of Analysis of Oligonucleotides and Related Products

Author : Jose V. Bonilla
Publisher : CRC Press
ISBN 13 : 1439819947
Total Pages : 502 pages
Book Rating : 4.4/5 (398 download)

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Book Synopsis Handbook of Analysis of Oligonucleotides and Related Products by : Jose V. Bonilla

Download or read book Handbook of Analysis of Oligonucleotides and Related Products written by Jose V. Bonilla and published by CRC Press. This book was released on 2011-02-23 with total page 502 pages. Available in PDF, EPUB and Kindle. Book excerpt: Oligonucleotides represent one of the most significant pharmaceutical breakthroughs in recent years, showing great promise as diagnostic and therapeutic agents for malignant tumors, cardiovascular disease, diabetes, viral infections, and many other degenerative disorders. The Handbook of Analysis of Oligonucleotides and Related Products is an essen

Revival

Author : Saghir Akhtar
Publisher : CRC Press
ISBN 13 : 9781138558571
Total Pages : 318 pages
Book Rating : 4.5/5 (585 download)

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Book Synopsis Revival by : Saghir Akhtar

Download or read book Revival written by Saghir Akhtar and published by CRC Press. This book was released on 2019-01-25 with total page 318 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.

Antisense Research and Application

Author : Stanley T. Crooke
Publisher : Springer Science & Business Media
ISBN 13 : 3642587852
Total Pages : 646 pages
Book Rating : 4.6/5 (425 download)

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Book Synopsis Antisense Research and Application by : Stanley T. Crooke

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.

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Author : Nicola Brunetti-Pierri
Publisher : Springer
ISBN 13 : 3319534572
Total Pages : 220 pages
Book Rating : 4.3/5 (195 download)

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Book Synopsis Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders by : Nicola Brunetti-Pierri

Download or read book Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders written by Nicola Brunetti-Pierri and published by Springer. This book was released on 2017-05-22 with total page 220 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields.

Therapeutic Oligonucleotides

Author : Jens Kurreck
Publisher : Royal Society of Chemistry
ISBN 13 : 1847558275
Total Pages : 362 pages
Book Rating : 4.8/5 (475 download)

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Book Synopsis Therapeutic Oligonucleotides by : Jens Kurreck

Download or read book Therapeutic Oligonucleotides written by Jens Kurreck and published by Royal Society of Chemistry. This book was released on 2008-04-22 with total page 362 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a comprehensive overview of the development of therapeutic oligonucleotides for therapeutic applications, touching on a number of additional oligonucleotides including a number of small interfering RNAs currently in various phases of clinical development. Written by leading expert scientists from both academia and leading biotechnical companies, the authors provide a compelling update on current status of RNA interference with emphasis on fascinating topics including oligonucleotides: antisense oligonucleotides, ribozymes, siRNAs, decoy oligonucleotides and aptamers. This exceptional work will be a valid resource for researchers and students as well as academia, consultants and scientists.