Oligonucleotide Based Drugs And Therapeutics

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Oligonucleotide-Based Drugs and Therapeutics

Nicolay Ferrari,Rosanne Seguin
Oligonucleotide Based Drugs and Therapeutics Book PDF

Author : Nicolay Ferrari,Rosanne Seguin
Publisher : John Wiley & Sons
Page : 576 pages
File Size : 46,9 Mb
Release : 2018-06-06
Category : Medical
ISBN : 9781119070290

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Oligonucleotide-Based Drugs and Therapeutics by Nicolay Ferrari,Rosanne Seguin Pdf

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

Olof Gissberg,Rula Zain,Karin E. Lundin
Oligonucleotide Based Therapies Book PDF

Author : Olof Gissberg,Rula Zain,Karin E. Lundin
Publisher : Humana
Page : 344 pages
File Size : 40,5 Mb
Release : 2020-08-15
Category : Medical
ISBN : 149399672X

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Oligonucleotide-Based Therapies by Olof Gissberg,Rula Zain,Karin E. Lundin Pdf

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.

Antisense Drug Technology

Stanley T. Crooke
Antisense Drug Technology Book PDF

Author : Stanley T. Crooke
Publisher : CRC Press
Page : 848 pages
File Size : 55,9 Mb
Release : 2007-07-25
Category : Medical
ISBN : 9781439832509

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Antisense Drug Technology by Stanley T. Crooke Pdf

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

Therapeutic Oligonucleotides

Jens Kurreck
Therapeutic Oligonucleotides Book PDF

Author : Jens Kurreck
Publisher : Royal Society of Chemistry
Page : 362 pages
File Size : 46,8 Mb
Release : 2008
Category : Medical
ISBN : 9780854041169

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Therapeutic Oligonucleotides by Jens Kurreck Pdf

This book provides a compelling overall update on current status of RNA interference

Antisense Research and Applications

Stanley T. Crooke,Bernard Lebleu
Antisense Research and Applications Book PDF

Author : Stanley T. Crooke,Bernard Lebleu
Publisher : CRC Press
Page : 620 pages
File Size : 44,8 Mb
Release : 1993-05-27
Category : Medical
ISBN : 084934705X

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Antisense Research and Applications by Stanley T. Crooke,Bernard Lebleu Pdf

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.

Synthesis of Therapeutic Oligonucleotides

Satoshi Obika,Mitsuo Sekine
Synthesis of Therapeutic Oligonucleotides Book PDF

Author : Satoshi Obika,Mitsuo Sekine
Publisher : Springer
Page : 284 pages
File Size : 43,6 Mb
Release : 2018-12-08
Category : Medical
ISBN : 9789811319129

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Synthesis of Therapeutic Oligonucleotides by Satoshi Obika,Mitsuo Sekine Pdf

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.

Targeting of Drugs 5

Gregory Gregoriadis,Brenda McCormack
Targeting of Drugs 5 Book PDF

Author : Gregory Gregoriadis,Brenda McCormack
Publisher : Springer Science & Business Media
Page : 201 pages
File Size : 55,6 Mb
Release : 2012-12-06
Category : Medical
ISBN : 9781461564058

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Targeting of Drugs 5 by Gregory Gregoriadis,Brenda McCormack Pdf

Proceedings of a NATO ASI held in Cape Sounion, Greece, June 24-July 5, 1995

Advances in Nucleic Acid Therapeutics

Michael J Gait,Sudhir Agrawal
Advances in Nucleic Acid Therapeutics Book PDF

Author : Michael J Gait,Sudhir Agrawal
Publisher : Royal Society of Chemistry
Page : 566 pages
File Size : 54,9 Mb
Release : 2019-02-11
Category : Science
ISBN : 9781788012096

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Advances in Nucleic Acid Therapeutics by Michael J Gait,Sudhir Agrawal Pdf

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.

Delivery Strategies for Antisense Oligonucleotide Therapeutics

Saghir Akhtar
Delivery Strategies for Antisense Oligonucleotide Therapeutics Book PDF

Author : Saghir Akhtar
Publisher : CRC Press
Page : 393 pages
File Size : 46,8 Mb
Release : 2017-07-28
Category : Medical
ISBN : 9781351366137

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Delivery Strategies for Antisense Oligonucleotide Therapeutics by Saghir Akhtar Pdf

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.

Oligonucleotides as Therapeutic Agents

Derek J. Chadwick,Gail Cardew
Oligonucleotides as Therapeutic Agents Book PDF

Author : Derek J. Chadwick,Gail Cardew
Publisher : John Wiley & Sons
Page : 260 pages
File Size : 50,9 Mb
Release : 2008-04-30
Category : Science
ISBN : 9780470515402

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Oligonucleotides as Therapeutic Agents by Derek J. Chadwick,Gail Cardew Pdf

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.

Antisense Research and Application

Stanley T. Crooke
Antisense Research and Application Book PDF

Author : Stanley T. Crooke
Publisher : Springer Science & Business Media
Page : 646 pages
File Size : 41,8 Mb
Release : 2012-12-06
Category : Medical
ISBN : 9783642587856

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Antisense Research and Application by Stanley T. Crooke Pdf

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.

Polyglutamine Disorders

Clévio Nóbrega,Luís Pereira de Almeida
Polyglutamine Disorders Book PDF

Author : Clévio Nóbrega,Luís Pereira de Almeida
Publisher : Springer
Page : 469 pages
File Size : 48,6 Mb
Release : 2018-02-09
Category : Medical
ISBN : 9783319717791

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Polyglutamine Disorders by Clévio Nóbrega,Luís Pereira de Almeida Pdf

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.

Antisense Therapeutics

M. Ian Phillips
Antisense Therapeutics Book PDF

Author : M. Ian Phillips
Publisher : Humana Press
Page : 344 pages
File Size : 41,9 Mb
Release : 2004-09-16
Category : Science
ISBN : 1588292053

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Antisense Therapeutics by M. Ian Phillips Pdf

A timely primer on the new reality of antisense and RNA inhibition for treating a broad range of diseases. The authors show how antisense oligonucleotides are being designed and studied in relation to hypertension, various cancers, inflammatory bowel disease, brain disorders, the blood-brain barrier, and drug delivery. Highlights include RNA-based therapies for many diseases, up-to-date methods and applications, and insight into the enormous potential to provide a new generation of drugs.

Nucleic Acid Drugs

Akira Murakami
Nucleic Acid Drugs Book PDF

Author : Akira Murakami
Publisher : Springer
Page : 180 pages
File Size : 52,5 Mb
Release : 2012-07-04
Category : Technology & Engineering
ISBN : 9783642304637

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Nucleic Acid Drugs by Akira Murakami Pdf

New Antisense Strategies: Chemical Synthesis of RNA Oligomers, by Junichi Yano und Gerald E. Smyth Development and Modification of Decoy Oligodeoxynucleotides for Clinical Application, by Mariana Kiomy Osako, Hironori Nakagami und Ryuichi Morishita Modulation of Endosomal Toll-Like Receptor-Mediated Immune Responses by Synthetic Oligonucleotides, by Ekambar R. Kandimalla und Sudhir Agrawal Delivery of Nucleic Acid Drugs, by Yan Lee und Kazunori Kataoka Aptamer: Biology to Applications, by Yoshikazu Nakamura Development and Clinical Applications of Nucleic Acid Therapeutics, by Veenu Aishwarya, Anna Kalota und Alan M. Gewirtz

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Nicola Brunetti-Pierri
Safety and Efficacy of Gene Based Therapeutics for Inherited Disorders Book PDF

Author : Nicola Brunetti-Pierri
Publisher : Springer
Page : 220 pages
File Size : 44,6 Mb
Release : 2017-05-22
Category : Medical
ISBN : 9783319534572

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Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders by Nicola Brunetti-Pierri Pdf

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.