RNA Interference from Biology to Therapeutics

This volume addresses the role of different RNAi molecules in cellular processes as rational for diagnostic and therapeutic approaches. It covers RNAi therapeutic design to optimize siRNA potency and reduce off-target effects.

The enormous potential of siRNA as a therapeutic has led to an explosion of interest from the scientific community. There has been intense interest from Big Pharma to capitalise on this new technology but the fact remains that delivery is a key determinant in realizing the full clinical potential of RNA interference. There is an urgent need for better delivery methods to take this technology forward. This book addresses the role of different RNAi molecules in cellular processes as rational for diagnostic and therapeutic approaches. This book will cover RNAi therapeutic design to optimize siRNA potency and reduce off-target effects and current delivery technologies to overcome both intracellular and extracellular barriers. The reader will gain an insight into RNA interference from the cellular mechanisms to screening to siRNA design right through to diagnostic and therapeutic applications.

RNA Interference Pathways and Therapeutic Exploitation RNA Interference. - Synthetic Dicer-Substrate siRNAs as Triggers of RNA Interference. - The Role of MicroRNAs in Natural Tissue Development and Application in Regenerative Medicine. - Intracellular Delivery Considerations for RNAi Therapeutics. - Mucosal Delivery of RNAi Therapeutics. - Nanomedicines for Systemic Delivery of RNAi Therapeutics. - Lipidoids: A Combinatorial Approach to siRNA Delivery. - Polymeric Micelles for siRNA Delivery. - RNAi Therapeutic Delivery by Exosomes. - Aptamer-Mediated siRNA Targeting. - RNAi as Anti-Viral Therapy: The HIV-1 Case. - Genome-Wide RNAi Screen for Identification of Human Host Factors Crucial for Influenza Virus Replication. - The Application of microRNAs in Cancer Diagnostics. - Therapeutic Application of miRNAs in Cancer. - Clinical Development of an Inhalable Anti-RSV siRNA Treatment.