Main description:

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.

Contents:

1. RNA Interference: Story and Mechanisms

Mouldy Sioud

2. Selection of Chemical Modifications in the siRNA Seed Region That Repress Off-target Effect

Yoshiaki Kobayashi, Kengo Miyamoto, Misako Aida, and Kumiko Ui-Tei

3. Stick-based Methods for Aptamer-mediated siRNA Targeted Delivery

Silvia Catuogno, Carla Lucia Esposito, and Paloma H. Giangrande

4. Evaluation of siRNA Stability and Interaction With Serum Components Using an Agarose Gel-Based Single-Molecule FRET Labeling Method

Martina Tuttolomondo and Henrik J. Ditzel

5. Development of siRNA Therapeutics for the Treatment of Liver Diseases Anja Holm, Marianne Bengtson Lovendorf, and Sakari Kauppinen

6. siRNA Design and GalNAc-Empowered Hepatic Targeted Delivery

Mei Lu, Mengjie Zhang, Bo Hu, and Yuanyu Huang

7. Synthesis of GalNAc-Oligonucleotide Conjugates Using GalNAc Phosphoramidite and Triple-GalNAc CPG Solid Support

Egor A. Ulashchik, Yury V. Martynenko-Makaev, Tatsiana P. Akhlamionok, Denis M. Melnik, Vadim V. Shmanai, and Timofei S. Zatsepin

8. Preparation of Lipid-Conjugated siRNA Oligonucleotides for Enhancing Gene Inhibition in Mammalian Cells

Santiago Grijalvo, Sandra M. Ocampo,Jose Carlos Perales, and Ramon Eritja

9. Engineering of Solid Dosages Forms of siRNA-loaded Lipidoid-Polymer Hybrid Nanoparticles using a quality by Design Approach Abhijeet Lokras, Camilla Foged, and Aneesh Thakur

10. Preparation and Characterization of siRNA-loaded Liposomes

Yang Liu and Leaf Huang

11. SiRNA Vehicles for High Endosomal Escapability

Hiroyuki Koide, Sei Yonezawa, and Tomohiro Asai

12. SiRNA Design and Delivery Based on Carbon Nanotubes

Danyang Li and Khuloud T. Al-Jamal

13. MRI-Visible Nanovehicle for Efficient siRNA Delivery

Rongrong Jin, Wencheng Zhu, Gan Lin, Gang Liu, and Hua Ai

14. Cationic Dendrimers for siRNA Delivery: An Overview of Methods for in Vitro / In Vivo Characterization

Erik Laurini, Suzana Aulica, Domenico Marson, Maurizio Fermeglia, and Sabrina Pricl

15. ITC for Characterization of Self-Assembly Process of Cationic Dendrons for siRNA Delivery

Erik Laurini, Suzana Aulic, Natasa Skoko, Domenico Marson, Maurizio Fermeglia, Sabrina Pricl

16. Cationic Dendrimers for siRNA Delivery - Computational Approaches for Characterization

Domenico Marson, Suzana Aulic, Maurizio Fermeglia, Erik Laurini, and Sabrina Pricl

17. Fundamental and Practical Aspects in the Formulation of Colloidal Polyelectrolyte Complexes of Chitosan and siRNA

Tim Delas and Christophe Schatz

18. Cell-Penetrating Peptides Delivering siRNAs: An Overview

Luca Falato, Maxime Gestin, and UElo Langel

19. Non-Covalent Encapsulation of siRNA with Cell-penetrating Peptides Martina Tuttolomondo and Henrik J. Ditzel

20. siRNA Therapeutics for Protein Misfolding Diseases of the Central Nervous System

Mark D. Zabel, Luke Mollnow, and Heather Bender

21. siRNA Loaded-exosomes

Maria Izco and Lydia Alvarez-Erviti

22. Production and Characterization of Anti-CCR5 siRNA-Loaded Polycaprolactone Nanoparticles for Topical Pre-Exposure Prophylaxis

Rafaela Ribeiro, Bruno Sarmento, and Jose das Neves

23. siRNA Therapeutics in Ocular Diseases

Javier Moreno-Montanes, Anne-Marie Bleau, Tamara Martinez, Beatriz Vargas, Maria Victoria Gonzalez, and Ana Isabel Jimenez

24. Delivery of siRNA to the Eye: Protocol for a Feasibility Study to Assess Novel Delivery System for Topical Delivery of siRNA Therapeutics to the Ocular Surface

Paulina Baran-Rachwalska, Suzanne Saffie-Siebert, and C.B.Tara Moore