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Author: Patel Jayvadan Publisher: LAP Lambert Academic Publishing ISBN: 9783659201158 Category : Languages : en Pages : 100
Book Description
Over the past 2 decades, the antisense oligonucleotide technology has emerged as a valid approach to selectively modulate gene expression. By adhering to a strict set of specific rules, ongoing in vitro studies using antisense oligonucleotides permit the characterization of new targets and new potential therapeutic compounds. The number of in vitro experiments has increased continuously, and this has led to numerous therapeutic trials, a few of which now appear preliminarily to be positive. However, the optimal use of antisense oligonucleotides in the treatment of disease requires the resolution of problems relating to effective design, enhanced biological activity, and efficient target delivery. These issues are currently being actively addressed and will hopefully continue to shed light on ways to increase therapeutic efficacy and specificity.
Author: Patel Jayvadan Publisher: LAP Lambert Academic Publishing ISBN: 9783659201158 Category : Languages : en Pages : 100
Book Description
Over the past 2 decades, the antisense oligonucleotide technology has emerged as a valid approach to selectively modulate gene expression. By adhering to a strict set of specific rules, ongoing in vitro studies using antisense oligonucleotides permit the characterization of new targets and new potential therapeutic compounds. The number of in vitro experiments has increased continuously, and this has led to numerous therapeutic trials, a few of which now appear preliminarily to be positive. However, the optimal use of antisense oligonucleotides in the treatment of disease requires the resolution of problems relating to effective design, enhanced biological activity, and efficient target delivery. These issues are currently being actively addressed and will hopefully continue to shed light on ways to increase therapeutic efficacy and specificity.
Author: Nicolay Ferrari Publisher: John Wiley & Sons ISBN: 1119070295 Category : Medical Languages : en Pages : 576
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: Saghir Akhtar Publisher: CRC Press ISBN: 1351366130 Category : Medical Languages : en Pages : 393
Book Description
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.
Author: Stanley T. Crooke Publisher: CRC Press ISBN: 1439832501 Category : Medical Languages : en Pages : 848
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: D. Robert Lu Publisher: Springer Science & Business Media ISBN: 1592597459 Category : Medical Languages : en Pages : 621
Book Description
An authoritative and up-to-date survey of the fundamental principles, and practice of drug delivery at the cellular level. On the principles side, the authors discuss the broad spectrum of cellular delivery, ranging from coverage of cell-mediated immunity, gene delivery, and protein targeting, to cellular drug transport, cellular drug permeability, and a variety of carrier system related to targeted drug delivery. On the practice side, the authors focus on technological developments in cellular drug delivery, including novel formulations for the delivery of DNA and antisense oligonucleotides ,as well as drug targeting with immunoglobulin formulations and antibody-mediated approaches.
Author: Clévio Nóbrega Publisher: Springer ISBN: 3319717790 Category : Medical Languages : en Pages : 467
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: Olof Gissberg Publisher: Humana ISBN: 9781493996728 Category : Medical Languages : en Pages : 344
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: Akira Murakami Publisher: Springer ISBN: 364230463X Category : Technology & Engineering Languages : en Pages : 189
Book Description
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
Author: Patel Jayvadan
Author: Patel Jayvadan
Author: Nicolay Ferrari
Author: Jens Kurreck
Author: Saghir Akhtar
Author: Stanley T. Crooke
Author: Gregory Gregoriadis
Author: D. Robert Lu
Author: Clévio Nóbrega
Author: Olof Gissberg
Author: Akira Murakami