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 Delivery Nonclinical Safety Assessments and Clinical Pharmacokinetics: A Regulatory Perspective Bioconjugation of siRNA for Site Specifi c Delivery Nanoscale Delivery Systems for RNAi Environmentally-Responsive Delivery Systems for RNAi Light-sensitive RNAi Viral-Mediated Delivery of shRNA and miRNA RNAi applications in Cancer Therapy, Liver Diseases, Hepatitis B, and Ocular Disease miRNA as therapeutic agents and targets Advanced 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.

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Mahato I. Ram Advanced Delivery and Therapeutic Applications of RNAi