RNA Interference

RNA Interference: Application to Drug Discovery and Challenges to Pharmaceutical Development provides a general overview of this rapidly emerging field, with a strong emphasis on issues and aspects that are important to a drug development team. The first part covers more general background of RNA interference and its application in drug discovery. In the second part, the book addresses siRNA (small interfering RNA), a pharmaceutically potent form, and its use and delivery in therapeutics along with manufacturing and delivery aspects. CONTRIBUTORS.

PREFACE.

FOREWORD.

I. Biology of RNA Interference.

1 RNA Interference: What is it? (James A. Birchler).

2 Nucleic Acids as Regulatory Molecules (Faqing Huang, C. J. Yu, and Yanlin Guo).

3 Use of siRNA Oligonucleotides to Study Gene Function (Kristin A. Wiederholt and Adam N. Harris).

4 Genome Scanning by RNA Interference (Roderick L. Beijersbergen and Oliver C. Steinbach).

II Development of siRNA for Therapeutic Applications.

5 Discovery of New siRNA Delivery Agents (Paul H. Johnson, Diane Frank, Kunyuan Cui, Roger Adami, Harry Wang, Michael Houston, and Steven C. Quay).

6 siRNA Delivery Vehicles (Aliasger K. Salem and Mark A. Behlke).

7 Interferon, Cytokine Induction and Other Potential In Vivo Toxicities (Adam Judge, Marjorie Robbins, and Ian MacLachlan).

8 Application of RNA Interference to Viral Diseases (Ralph A. Tripp and Stephen Mark Tompkins).

Index.

"This is an accessible book that approaches the goal of therapeutic RNAi delivery from a technological viewpoint. Chapter organization and figure presentation allow for interrupted reading without disrupting the flow. Cell and molecular biologists will find both interesting details and practical methods to improve the preclinical applicability of a research project. While therapeutic RNAi is still largely in the development stage, this book promotes a mindset to continue the search for effective delivery strategies." (Doody's, 7 October 2011) PAUL H. JOHNSON, PhD, is a cofounder and Chief Scientific Officer at PhaseRx, Inc. He is the author of numerous scientific papers and patents, and was the recipient of the 2007 Jeffrey Wenig Memorial Award for Outstanding Achievement in Drug Delivery.

A general overview of RNA interference for the novice reader

Today, RNA molecules are used extensively in academic labs and the pharmaceutical industry to probe gene function and validate gene and protein targets for drug discovery. This book provides an overview of RNA interference for those unfamiliar with this rapidly emerging field. It covers mechanisms of action, its applications as a target validation and identification tool, toxicity concerns, and development of siRNA molecules as novel therapeutics.

Written by experts in the field, each chapter focuses on an important aspect of RNAi biology or therapeutic discovery and application. While various aspects of current research, important discoveries, technology description, and methodology are covered, the book focuses on identifying issues that must be overcome for the successful development of RNAi technology. With a strong emphasis on issues and aspects that are important to a drug development team, the book:

• Presents a general overview of RNA interference biology, along with a strong emphasis on therapeutic applications issues important for drug development

• Addresses siRNA (small interfering RNA), a pharmaceutically-potent form, and how it is used in therapeutics, along with toxicity and delivery aspects

• Emphasizes industrial applications such as chemical modifications and delivery

• Focuses on gene function validation and siRNA as a new class of therapeutics

• Includes pharmaceutical topics of discovery and development of delivery agents, toxicity, and viral disease applications

Clear, easy to understand, and focused on key issues for future research and development, RNA Interference is intended for non-specialist scientists, working in both academia and the pharmaceutical industry, who need to know and work within the dynamic area of RNA interference.