Design and Delivery of SiRNA Therapeutics [Paperback]

This volume details protocols on rationale design of therapeutic siRNA molecules and its encapsulation with smart vehicles to overcome the barriers to an effective administration in vivo. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls.

 Authoritative and cutting-edge, Design and Delivery of SiRNA Therapeutics aims to ensure successful results in the further study of this vital field.

RNA Interference: Story and Mechanisms.- Selection of Chemical Modifications in the siRNA Seed Region That Repress Off-target Effect.- Stick-based Methods for Aptamer-mediated siRNA Targeted Delivery.- Evaluation of siRNA Stability and Interaction With Serum Components Using an Agarose Gel-Based Single-Molecule FRET Labeling Method.- Development of siRNA Therapeutics for the Treatment of Liver Diseases.- siRNA Design and GalNAc-Empowered Hepatic Targeted Delivery.- Synthesis of GalNAc-Oligonucleotide Conjugates Using GalNAc Phosphoramidite and Triple-GalNAc CPG Solid Support.- Preparation of Lipid-Conjugated siRNA Oligonucleotides for Enhancing Gene Inhibition in Mammalian Cells.- Engineering of Solid Dosages Forms of siRNA-loaded LipidoidPolymer Hybrid Nanoparticles using a quality by Design Approach.- Preparation and Characterization of siRNA-loaded Liposomes.- SiRNA Vehicles for High Endosomal Escapability.- SiRNA Design andDelivery Based on Carbon Nanotubes.- MRI-Visible Nanovehicle for Efficient siRNA Delivery.- Cationic Dendrimers for siRNA Delivery: An Overview of Methods for in Vitro / In Vivl³Œ