Design and Delivery of SiRNA Therapeutics

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.

Includes cutting-edge methods and protocols Provides step-by-step detail essential for reproducible results Contains key notes and implementation advice from the experts

Inhalt

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 Vivo Characterization.- ITC for Characterization of Self-Assembly Process of Cationic Dendrons for siRNA Delivery.- Cationic Dendrimers for siRNA Delivery - Computational Approaches for Characterization.- Fundamental and Practical Aspects in the Formulation of Colloidal Polyelectrolyte Complexes of Chitosan and siRNA.- Cell-Penetrating Peptides Delivering siRNAs: An Overview.- Non-Covalent Encapsulation of siRNA with Cell-penetrating Peptides.- siRNA Therapeutics for Protein Misfolding Diseases of the Central Nervous System.- siRNA Loaded-exosomes.- Production and Characterization of Anti-CCR5 siRNA-Loaded Polycaprolactone Nanoparticles for Topical Pre-Exposure Prophylaxis.- siRNA Therapeutics in Ocular Diseases.- Delivery of siRNA to the Eye: Protocol fora Feasibility Study to Assess Novel Delivery System for Topical Delivery of siRNA Therapeutics to the Ocular Surface.