Advances in Nucleic Acid Therapeutics
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.
Advances in Nucleic Acid Therapeutics, The Royal Society of Chemistry, 2019.
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CHAPTER 1: History and Development of Nucleotide Analogues in Nucleic Acids Drugsp1-21ByMichael J. GaitMichael J. GaitMedical Research Council, Laboratory of Molecular BiologyFrancis Crick AvenueCambridge CB2 0QHUK[email protected]Search for other works by this author on:
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CHAPTER 2: Mechanisms of Antisense Oligonucleotidesp22-31ByZhongtian Liu;Zhongtian LiuDepartments of Pharmacology & Biochemistry, University of Texas Southwestern Medical Center6001 Forest Park RoadDallasTexas 75390-9041USA[email protected]Search for other works by this author on:David R. CoreyDavid R. CoreyDepartments of Pharmacology & Biochemistry, University of Texas Southwestern Medical Center6001 Forest Park RoadDallasTexas 75390-9041USA[email protected]Search for other works by this author on:
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CHAPTER 3: The Medicinal Chemistry of RNase H-activating Antisense Oligonucleotidesp32-61ByEric E. SwayzeEric E. SwayzeSearch for other works by this author on:
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CHAPTER 4: Antisense Technology: Liver Targeting and Beyond for Drug Discoveryp62-79ByBrett P. MoniaBrett P. MoniaSearch for other works by this author on:
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CHAPTER 5: Oligonucleotide-based Toll-like Receptor Antagonists and Therapeutic Applicationsp80-102BySudhir AgrawalSudhir AgrawalSearch for other works by this author on:
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CHAPTER 6: Splicing Modulation for Therapeuticsp103-125ByElena Daoutsali;Elena DaoutsaliDepartment of Human Genetics, Leiden University Medical CenterEinthovenweg 20, 2333 ZCLeidenThe Netherlands[email protected]Search for other works by this author on:Annemieke Aartsma-RusAnnemieke Aartsma-RusDepartment of Human Genetics, Leiden University Medical CenterEinthovenweg 20, 2333 ZCLeidenThe Netherlands[email protected]Search for other works by this author on:
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CHAPTER 7: Targeting Toxic Repeatsp126-150ByM. Leontien van der Bent;M. Leontien van der BentRadboud Institute for Molecular Life Sciences (RIMLS), Radboud University Medical Center, Department of Cell BiologyGeert Grooteplein 26-286525 GA NijmegenThe Netherlands[email protected]Radboud Institute for Molecular Life Sciences (RIMLS), Radboud University Medical Center, Department of BiochemistryGeert Grooteplein 26-286525 GA NijmegenThe NetherlandsSearch for other works by this author on:Remco T. P. van Cruchten;Remco T. P. van CruchtenRadboud Institute for Molecular Life Sciences (RIMLS), Radboud University Medical Center, Department of Cell BiologyGeert Grooteplein 26-286525 GA NijmegenThe Netherlands[email protected]Search for other works by this author on:Derick G. WansinkDerick G. WansinkRadboud Institute for Molecular Life Sciences (RIMLS), Radboud University Medical Center, Department of Cell BiologyGeert Grooteplein 26-286525 GA NijmegenThe Netherlands[email protected]Search for other works by this author on:
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CHAPTER 8: Research and Development of Oligonucleotides Targeting MicroRNAs (miRNAs)p151-180ByA. L. Malinowska;A. L. MalinowskaETH Zürich, Department of Chemistry and Applied BiosciencesHCI H 437, Vladimir-Prelog-Weg 1-5/108093 ZürichSwitzerland[email protected]Search for other works by this author on:J. HallJ. HallETH Zürich, Department of Chemistry and Applied BiosciencesHCI H 437, Vladimir-Prelog-Weg 1-5/108093 ZürichSwitzerland[email protected]Search for other works by this author on:
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CHAPTER 9: Oligonucleotide Targeting of Long Non-coding RNAsp181-205ByI. Blokhin;I. BlokhinCenter for Therapeutic Innovation and Department of Psychiatry and Behavioral Sciences, University of Miami, Miller School of Medicine1501 NW 10th AvenueMiamiFL 33136USA[email protected]Search for other works by this author on:O. Khorkova;O. KhorkovaOPKO Health Inc.10320 USA Today WayMiramarFL 33025USASearch for other works by this author on:J. Hsiao;J. HsiaoOPKO Health Inc.10320 USA Today WayMiramarFL 33025USASearch for other works by this author on:C. WahlestedtC. WahlestedtCenter for Therapeutic Innovation and Department of Psychiatry and Behavioral Sciences, University of Miami, Miller School of Medicine1501 NW 10th AvenueMiamiFL 33136USA[email protected]Search for other works by this author on:
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CHAPTER 10: Conjugate-mediated Delivery of RNAi-based Therapeutics: Enhancing Pharmacokinetics–Pharmacodynamics Relationships of Medicinal Oligonucleotidesp206-232ByBruno M. D. C. Godinho;Bruno M. D. C. GodinhoRNA Therapeutics Institute, University of Massachusetts Medical SchoolWorcesterMAUSA[email protected]Search for other works by this author on:Andrew H. Coles;Andrew H. ColesRNA Therapeutics Institute, University of Massachusetts Medical SchoolWorcesterMAUSA[email protected]Search for other works by this author on:Anastasia KhvorovaAnastasia KhvorovaRNA Therapeutics Institute, University of Massachusetts Medical SchoolWorcesterMAUSA[email protected]Program in Molecular Medicine, University of Massachusetts Medical SchoolWorcesterMAUSASearch for other works by this author on:
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CHAPTER 11: Liver-targeted RNAi Therapeutics: Principles and Applicationsp233-265ByMuthiah ManoharanMuthiah ManoharanSearch for other works by this author on:
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CHAPTER 12: Advances and Challenges of RNAi-Based Anti-HIV Therapeuticsp266-291ByJ. Zhou;J. ZhouDepartment of Molecular and Cellular Biology, Beckman Research Institute of City of Hope1500 E. Duarte RdDuarteCA 91010USASearch for other works by this author on:J. J. RossiJ. J. RossiDepartment of Molecular and Cellular Biology, Beckman Research Institute of City of Hope1500 E. Duarte RdDuarteCA 91010USAIrell and Manella Graduate School of Biological Sciences, Beckman Research Institute of City of Hope1500 E. Duarte RdDuarteCA 91010USA[email protected]Search for other works by this author on:
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CHAPTER 13: Nucleic Acid Innate Immune Receptorsp292-305ByShin-Ichiroh Saitoh;Shin-Ichiroh SaitohDivision of Innate Immunity, Department of Microbiology and Immunology, The Institute of Medical Science, The University of Tokyo4-6-1 ShirokanedaiMinatokuTokyo 108-8639Japan[email protected]Search for other works by this author on:Kensuke MiyakeKensuke MiyakeDivision of Innate Immunity, Department of Microbiology and Immunology, The Institute of Medical Science, The University of Tokyo4-6-1 ShirokanedaiMinatokuTokyo 108-8639Japan[email protected]Search for other works by this author on:
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CHAPTER 14: Synthetic Agonists of Toll-like Receptors and Therapeutic Applicationsp306-338ByEkambar R. KandimallaEkambar R. KandimallaSearch for other works by this author on:
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CHAPTER 15: Prostate-specific Membrane Antigen (PSMA) Aptamers for Prostate Cancer Imaging and Therapyp339-366ByShambhavi Shubham;Shambhavi ShubhamDepartment of Internal Medicine, University of IowaIowa CityIA52242USASearch for other works by this author on:Li-Hsien Lin;Li-Hsien LinDepartment of Internal Medicine, University of IowaIowa CityIA52242USASearch for other works by this author on:Ofonime Udofot;Ofonime UdofotDepartment of Internal Medicine, University of IowaIowa CityIA52242USAAbboud Cardiovascular Research Center, University of IowaIowa CityIA 52242USASearch for other works by this author on:Sven Krupse;Sven KrupseDepartment of Internal Medicine, University of IowaIowa CityIA52242USASearch for other works by this author on:Paloma H. GiangrandePaloma H. GiangrandeDepartment of Internal Medicine, University of IowaIowa CityIA52242USADepartment of Radiation Oncology University of IowaIowa CityIA52242USAMolecular & Cellular Biology Program, University of IowaIowa CityIA52242USAMedical Scientist Training Program, University of IowaIowa CityIA 52242USAAbboud Cardiovascular Research Center, University of IowaIowa CityIA 52242USAHolden Comprehensive Cancer Center, University of IowaIowa CityIAUSAEnvironmental Health Sciences Research Center (EHSRC), University of IowaIowa CityIAUSA[email protected]Search for other works by this author on:
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CHAPTER 16: Aptamers and Clinical Applicationsp367-399ByG. ZonG. ZonSearch for other works by this author on:
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CHAPTER 17: CRISPR-based Technologies for Genome Engineering: Properties, Current Improvements and Applications in Medicinep400-433BySylvain Geny;Sylvain GenyMuseum National d'Histoire Naturelle, INSERM U1154, CNRS UMR 7196, Sorbonne Universités43 rue CuvierParis F-75231France[email protected]Search for other works by this author on:Elaheh Sadat Hosseini;Elaheh Sadat HosseiniMuseum National d'Histoire Naturelle, INSERM U1154, CNRS UMR 7196, Sorbonne Universités43 rue CuvierParis F-75231France[email protected]Search for other works by this author on:Jean-Paul Concordet;Jean-Paul ConcordetMuseum National d'Histoire Naturelle, INSERM U1154, CNRS UMR 7196, Sorbonne Universités43 rue CuvierParis F-75231France[email protected]Search for other works by this author on:Carine GiovannangeliCarine GiovannangeliMuseum National d'Histoire Naturelle, INSERM U1154, CNRS UMR 7196, Sorbonne Universités43 rue CuvierParis F-75231France[email protected]Search for other works by this author on:
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CHAPTER 18: Therapeutic Potential of Ribozymesp434-452ByDarko Balke;Darko BalkeUniversity of Greifswald, Institute of BiochemistryFelix-Hausdorff-Str. 417487 GreifswaldGermany[email protected]Search for other works by this author on:Sabine MüllerSabine MüllerUniversity of Greifswald, Institute of BiochemistryFelix-Hausdorff-Str. 417487 GreifswaldGermany[email protected]Search for other works by this author on:
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CHAPTER 19: Large-scale Automated Synthesis of Therapeutic Oligonucleotides: A Status Updatep453-473ByYogesh S. SanghviYogesh S. SanghviSearch for other works by this author on:
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CHAPTER 20: Preclinical and Clinical Drug-metabolism, Pharmacokinetics and Safety of Therapeutic Oligonucleotidesp474-531ByPatrik Andersson;Patrik AnderssonAdvanced Medicines Safety, Drug Safety and Metabolism, IMED Biotech UnitAstraZeneca, Pepparedsleden 1431 83 MölndalGothenburgSweden[email protected]Search for other works by this author on:Cathaline den BestenCathaline den BestenProQR Therapeutics BVZernikedreef 92333 CK LeidenThe NetherlandsSearch for other works by this author on:
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