Advanced Delivery and Therapeutic Applications of RNAi

Kun Cheng 지음

Wiley 2013년 04월 19일 출간

작품소개

책소개

RNA interference (RNAi) is a process in living cells whereby small double stranded RNA interferes with the expression of specific genes with complementary nucleotide sequence. Like many nucleic acid-based therapies, RNAi has great potential in treating various life-threatening diseases. However, the poor stability and cellular uptake of RNAi molecules remain considerable barriers to their efficient delivery which is paramount to a successful therapy. This book provides a comprehensive introduction to RNAi delivery, then goes on to discuss different delivery strategies, and concludes with current applications in various diseases. It covers:Mechanisms, Barriers, and Analysis of RNAi DeliveryNonclinical Safety Assessments and Clinical Pharmacokinetics: A Regulatory PerspectiveBioconjugation of siRNA for Site Specifi c DeliveryNanoscale Delivery Systems for RNAiEnvironmentally-Responsive Delivery Systems for RNAiLight-sensitive RNAiViral-Mediated Delivery of shRNA and miRNARNAi applications in Cancer Therapy, Liver Diseases, Hepatitis B, and Ocular DiseasemiRNA as therapeutic agents and targetsAdvanced Delivery and Therapeutic Applications of RNAi combines the essential aspects of developing RNAi therapeutics from bench to clinic, and is invaluable for researchers working on RNAi, drug discovery and delivery, biomedical engineering, biomaterials, molecular biology, and biotechnology.

목차

Preface xviiContributors xixAbout the Editors xxiiiPart 1 Introduction and Basics of RNAi 11 Mechanisms and Barriers to RNAi Delivery 3 Jiehua Zhou and John J. Rossi1.1 Introduction 31.2 Barriers to Systemic RNAi Delivery 51.3 Rational Design to Improve RNAi Efficacy 61.4 Chemical Modifications to Enhance siRNA Stability and Reduce Immune Response 71.5 Cellular Uptake and Intracellular Release of siRNA 71.6 Combinatorial Targeting for Targeted RNAi Delivery 81.7 Cell-Specific Aptamer-Functionalized Nanocarriers for RNAi Delivery 91.8 The Clinical Development and Challenges of siRNAs Therapeutics 101.9 Conclusion and Perspectives 12References 122 Analysis of siRNA Delivery Using Various Methodologies 19 Yi Pei2.1 Introduction 192.2 Checkpoints for Analyzing siRNA Delivery 202.3 Methods for Analysis of siRNA 262.4 Case Study for siRNA Delivery Analysis 38References 393 Challenges and Opportunities in Bringing RNAi Technologies from Bench to Bed 45 Sandesh Subramanya and Lance Ford3.1 Introduction 453.2 RNAi Mediator (siRNA or shRNA) 453.3 Safety Issues of RNAi Mediators 503.4 Efficacy of RNAi Mediators 523.5 RNAi Mediators in Clinical Trials 533.6 Conclusion 54References 55Nonclinical Safety Assessments and Clinical Pharmacokinetics for Oligonucleotide Therapeutics: A Regulatory Perspective 63 Shwu-Luan Lee, Paul Brown, Jian Wang and Robert T. Dorsam4.1 Introduction 634.2 Unique Properties of Oligonucleotide-based Therapeutics 634.3 Regulation of Oligonucleotide-Based Therapeutics 654.4 Conclusion 79Disclaimer 79 Appendix 79 References 80Role of Promoters and MicroRNA Backbone for Efficient Gene Silencing 83 <

작가정보

저자(글) Kun Cheng