Advanced Delivery and Therapeutic Applications of RNAi

By: Kun Cheng and Ram I. Mahato

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Synopsis: 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.