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Title Advances in nucleic acid therapeutics / edited by Sudhir Agrawal and Michael J. Gait.

Publication Info. Cambridge, UK : Royal Society of Chemistry, [2019]

Item Status

Description 1 online resource (1 volume).
Physical Medium polychrome
Description text file
Series Drug discovery series ; no. 68, 2041-3203
RSC drug discovery series ; 68. 2041-3203
Bibliography Includes bibliographical references and index.
Contents Intro; Half Title; Series Editors; Title; Copyright; Foreword; Preface; Contents; Chapter 1 History and Development of Nucleotide Analogues in Nucleic Acids Drugs 1; 1.1 Introduction; 1.2 The Antisense Concept; 1.3 Developments in Oligonucleotide Synthesis; 1.4 Choices for Antisense Oligodeoxynucleotide Modifications; 1.4.1 Backbone Modifications; 1.4.2 Heterocyclic Bases; 1.4.3 Sugar Modifications; 1.5 Gapmers Using Combinations of Modified Oligodeoxy and/or Oligoribonucleotides; 1.6 Antisense Conjugates; 1.7 The Role of Innate Immune Receptors in Nucleic Acid Therapeutics
1.8 Future DirectionsAcknowledgements; References; Chapter 2 Mechanisms of Antisense Oligonucleotides 22; 2.1 Introduction; 2.2 RNase H and ASO Action; 2.3 ASOs and Regulation of Splicing; 2.4 ASOs and Activation of Frataxin, a Case Study for an Emerging Mechanism; 2.5 Summary; Acknowledgements; References; Chapter 3 The Medicinal Chemistry of RNase H-activating Antisense Oligonucleotides 32; 3.1 Introduction to Gapmers; 3.2 Human RNase H1; 3.2.1 Biochemistry of Human RNase H1; 3.2.2 Structural Biology of Human RNase H1; 3.3 Structure-Activity Relationships of Gap Modifications
3.5.4 ASO Gapmer Duplexes3.6 Control of Protein Binding; 3.6.1 Interaction of Gapmer ASOs with Plasma Proteins; 3.6.2 Interaction of Gapmer-ASOs with Cell-surface Proteins; 3.6.3 Targeting Cell-surface Proteins for Cell-specific Delivery of Gapmers; 3.6.4 Avoiding Interactions with TLR Receptors to Avoid Immune-stimulatory Toxicities; 3.6.5 Optimizing Intracellular Distribution; 3.7 Conclusions; References; Chapter 4 Antisense Technology: Liver Targeting and Beyond for Drug Discovery 62; 4.1 Introduction; 4.2 Liver Targeting; 4.2.1 The Beginning; 4.2.2 Broadening the Liver-targeting Pipeline
4.3 Innovations in Liver Targeting4.4 Beyond the Liver; 4.5 Conclusions; References; Chapter 5 Oligonucleotide-based Toll-like Receptor Antagonists and Therapeutic Applications 80; 5.1 Introduction; 5.2 Oligonucleotide-based TLR Antagonists; 5.2.1 Structure-Activity Relationship Studies; 5.2.2 TLR Antagonists; 5.2.3 Inhibitory Activity of TLR Antagonists; 5.2.4 Inhibitory Activity of Clinical Candidates; 5.3 Studies of TLR Antagonists in Disease Models; 5.3.1 Psoriasis; 5.3.2 Systemic Lupus Erythematosus (SLE); 5.3.3 Rheumatoid Arthritis (RA); 5.3.4 Duchenne Muscular Dystrophy (DMD)
Summary Advances in Nucleic Acid Therapeutics provides a broad overview of techniques of contemporary interest in drug discovery.
Local Note eBooks on EBSCOhost EBSCO eBook Subscription Academic Collection - North America
Subject Nucleic acids -- Therapeutic use.
Nucleic acids -- Therapeutic use.
Nucleic acids.
Genre/Form Electronic books.
Added Author Agrawal, Sudhir, editor.
Gait, M. J. (Michael J.), editor.
Royal Society of Chemistry (Great Britain), issuing body.
Other Form: Print version: ADVANCES IN NUCLEIC ACID THERAPEUTICS. [Place of publication not identified], ROYAL Society OF CHEMISTR, 2019 1788012097 (OCoLC)1036274247
ISBN 9781788017329 (electronic book)
1788017323 (electronic book)
1788012097
9781788012096
9781788015714 (PDF ebook)
1788015711