| Description |
1 online resource (535 pages) |
| Contents |
Advanced Delivery and Therapeutic Applications of RNAi; Contents; Preface; Contributors; About the Editors; Part 1: Introduction and Basics of RNAi; 1 Mechanisms and Barriers to RNAi Delivery; 1.1 Introduction; 1.2 Barriers to Systemic RNAi Delivery; 1.3 Rational Design to Improve RNAi Efficacy; 1.4 Chemical Modifications to Enhance siRNA Stability and Reduce Immune Response; 1.5 Cellular Uptake and Intracellular Release of siRNA; 1.6 Combinatorial Targeting for Targeted RNAi Delivery; 1.7 Cell-Specific Aptamer-Functionalized Nanocarriers for RNAi Delivery |
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1.8 The Clinical Development and Challenges of siRNAs Therapeutics1.9 Conclusion and Perspectives; References; 2 Analysis of siRNA Delivery Using Various Methodologies; 2.1 Introduction; 2.2 Checkpoints for Analyzing siRNA Delivery; 2.2.1 Circulation Checkpoint; 2.2.2 Organ or Tissue Checkpoint; 2.2.3 Cellular Checkpoint; 2.2.4 RISC Checkpoint; 2.2.5 Target mRNA Knockdown (Indirect Checkpoint); 2.2.6 Protein and Outcome (Indirect Checkpoint); 2.2.7 Safety (Indirect Checkpoint); 2.3 Methods for Analysis of siRNA; 2.3.1 General Considerations; 2.3.2 Hybridization-Based (Non-Imaging) Methods |
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2.3.3 Non-Hybridization-Based (Non-Imaging) Methods2.3.4 Imaging-Based (Non-Hybridization) Methods; 2.3.5 Imaging-Based (Hybridization) Methods; 2.4 Case Study for siRNA Delivery Analysis; References; 3 Challenges and Opportunities in Bringing RNAi Technologies from Bench to Bed; 3.1 Introduction; 3.2 RNAi Mediator (siRNA or shRNA); 3.2.1 siRNA; 3.2.2 Vector-derived shRNA; 3.2.3 miRNAs; 3.3 Safety Issues of RNAi Mediators; 3.3.1 Immune Stimulation; 3.3.2 RNAi Overexpression; 3.4 Efficacy of RNAi Mediators; 3.4.1 Therapeutic Response; 3.5 RNAi Mediators in Clinical Trials; 3.6 Conclusion |
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5.2.1 Constitutive Promoters5.2.2 Inducible Promoters; 5.2.3 Site Specific Promoters; 5.3 miRNA-based shRNAs; 5.3.1 miRNA-based shRNA Enhances Gene Silencing; 5.3.2 miRNA-based shRNA Reduces Toxicities; 5.3.3 Application of miRNA-based shRNA for Combination Gene Therapy; 5.4 Concluding Remarks; References; Part 2: RNAi Delivery Strategies; 6 Bioconjugation of siRNA for Site-specific Delivery; 6.1 Introduction; 6.2 Conjugation Strategy; 6.2.1 RNA Chemical Modification; 6.2.2 Site of Conjugation; 6.2.3 Conjugation Chemistry; 6.3 Bioconjugates for Site-specific Delivery |
| Summary |
Commonly used by researchers to develop technologies for modifying and studying genetic process, RNA interference (RNAi) has many potential uses in medicine, biotechnology, and functional genomics. This book covers all essential aspects involved in the development of RNAi therapeutics, providing detailed guidance on the challenges and opportunities of bringing RNAi technologies from bench to clinic. It explores the design and mechanism of RNAi molecules, delivery strategies, and therapeutic applications in various diseases. Preclinical, regulatory, market, and intellectual aspects of RNAi tech |
| Bibliography |
References4 Nonclinical Safety Assessments and Clinical Pharmacokinetics for Oligonucleotide Therapeutics: A Regulatory Perspective; 4.1 Introduction; 4.2 Unique Properties of Oligonucleotide-based Therapeutics; 4.3 Regulation of Oligonucleotide-Based Therapeutics; 4.3.1 Submission to the FDA; 4.3.2 Review Process for Non-clinical Studies; 4.3.3 Regulatory Issues; 4.3.4 Clinical Pharmacokinetics; 4.4 Conclusion; Disclaimer; Appendix; References; 5 Role of Promoters and MicroRNA Backbone for Efficient Gene Silencing; 5.1 Introduction; 5.2 Promoters for shRNA Expression |
| Notes |
Print version record |
| Subject |
Science.
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RNA interference.
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RNA Interference
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RNA, Small Interfering -- therapeutic use
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Science
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sciences (philosophy)
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SCIENCE -- Chemistry -- Organic.
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RNA interference.
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Science.
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RNA -- Ús terapèutic.
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RNA -- Us terapeutic.
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| Form |
Electronic book
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| Author |
Mahato, Ram I
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| ISBN |
9781118610756 |
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111861075X |
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9781118610732 |
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1118610733 |
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9781119976868 |
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1119976863 |
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