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.

Autorentext

Kenneth Alan Howard is an Associate Professor and Group Leader at the Department of Molecular Biology at the Interdisciplinary Nanoscience Center (iNANO), Aarhus University, Denmark. His team is based in a highly interdisciplinary setting with research and teaching activities focused on advanced drug delivery, nanomedicine, RNA interference, and inflammation. Dr Howard is CEO of an Aarhus University spin-out company that provides delivery solutions for RNA interference-based therapeutics. Kenneth Alan Howard received a PhD in Pharmaceutical Science from the University of Nottingham, UK in 1995, and has held postdoctoral positions at the CRC Institute for Cancer Studies, University of Birmingham, UK and the School of Pharmacy, University of London. Dr Howard is an active member of the Controlled Release Society serving on the Board of Scientific Advisors (chair), Nominations Committee and organizer of a CRS Educational workshop RNA Interference Biology and Therapeutics.

Klappentext

The Nobel Prize winning discovery that small interfering RNA can be utilised to control cellular gene expression has propelled the field of RNA interference (RNAi) to the forefront of biomedical science as a potential molecular medicine set to revolutionalise disease treatment.

Harnessing the molecular mechanisms of RNAi and development of delivery technologies is crucial for its transformation into a therapeutic modality, this dependency is the focus of RNA Interference from Biology to Therapeutics that gives a comprehensive overview of RNAi biology and state-of-the-art delivery methods relevant to clinical translation of RNAi therapeutics.

Key players and shapers in the fields of RNAi and delivery science have been assembled in a single volume to produce a truly unique interdisciplinary text, making it a must-read for both students and experts in, and at the interface of, RNAi, pharmaceutical science and medicine. An attractive feature is the future perspectives section within each chapter that allows global leaders the opportunity to express their views on the direction the field is moving. Topics covered in the book include miRNA biology and therapeutic exploitation, exosome delivery and clinical translation. RNA Interference from Biology to Therapeutics is an up-to-the-minute, highly informative and invaluable text for those actively involved or interested in this fascinating and high-impact field.

Inhalt
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.