Therapeutic Applications of RNAi: Methods and Protocols

Overview

The phenomenon of RNA interference has rapidly moved from groundbreaking scientific discovery to promising therapeutic approach. However, even as RNAi-based drugs enter the clinic, significant challenges remain, particulary in the area of delivery. Therapeutic Applications of RNAi provides detailed prools in key areas of current focus, including testing of delivery vehicles, identification of appropriate model systems, and evaluation of the effects of RNAi in vivo. Produced by a team of internationally renowned ...

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Overview

The phenomenon of RNA interference has rapidly moved from groundbreaking scientific discovery to promising therapeutic approach. However, even as RNAi-based drugs enter the clinic, significant challenges remain, particulary in the area of delivery. Therapeutic Applications of RNAi provides detailed prools in key areas of current focus, including testing of delivery vehicles, identification of appropriate model systems, and evaluation of the effects of RNAi in vivo. Produced by a team of internationally renowned authors, the volume describes the therapeutic applications of RNAi and potential pitfalls in oncology, viral infections and CNS disease, using a variety of delivery methods, including liposomes, peptide-based nanoparticles, polycationic polymers, and viral vehicles. Written in the highly successful Methods in Molecular Biology™ series format, the chapters include brief introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory prools, and notes on troubleshooting and avoiding known pitfalls.

Authoritative and cutting-edge, Therapeutic Applications of RNAi is an ideal guide for scientists attempting to solve the numerous challenges in this field and revolutionize the treatment of disease.

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Editorial Reviews

Doody's Review Service
Reviewer: Ruben Mestril, PhD (Loyola University Medical Center)
Description: This volume in the Methods in Molecular Biology series is a compilation of laboratory protocols organized in 10 chapters dealing with the challenging task of delivering RNA interference molecules (RNAi) to live organisms to achieve therapeutic effects. Ever since the discovery in the 1990s that double-stranded RNA (dsRNA) is able to produce gene silencing, this approach has become the method of choice for gene expression knockdown. The potential therapeutic applications of RNAi to treat human disease are immense. The major hurdle, even today, in this rapidly expanding area of research is still the issue of delivery of RNAi to patients. The editors have recruited for this task a team of international experts from a number of different institutions. All of the chapters provide a succinct introduction to the topic, followed by the protocol with pertinent examples, and a notes section with useful troubleshooting suggestions.
Purpose: The main purpose is to concentrate on current protocols dealing with the delivery of RNAi using a diversity of methods. The book provides a comprehensive selection of approaches used to deliver RNAi to live organisms. It covers common approaches for delivery, such as viral vectors and liposomes, as well as the more cutting-edge ones such as using nanoparticles. This selection permits readers to focus on the method that best fits their target.
Audience: The book is written mainly for scientists, clinical researchers, and all investigators interested in the field of biotherapeutic research. It provides a diversity of methods to tackle the delivery of RNAi for the purpose of gene knockdown. Given the wide variety of approaches covered in this book, it is clear that most investigators will find something of interest.
Features: Current therapeutic approaches to delivery of RNAi in vivo to experimental animals are covered as a first step to discovering safe means of producing gene knockdown in patients. Among the most interesting chapters are chapter 1, "Development and Application of a Dual-Purpose Nanoparticle Platform for Delivery and Imaging of RNAi in Tumors," chapter 2, "Targeted Gene Silencing into Solid Tumors with Electrically Mediated siRNA Delivery," and chapter 6, "RNAi Using a Chitosan/siRNA Nanoparticle System: In Vitro and In Vivo Applications," all dedicated to the novel means of delivery of RNAi to live tissue. Also fascinating is the final chapter dedicated to the use of RNAi to control the splicing and expression of a trans gene. These techniques have the potential of providing important information that eventually will translate into valuable therapeutic applications in a wide diversity of human diseases.
Assessment: This volume in the series presents a good balance between the different approaches to RNAi delivery and it explores a number of potential solutions to the safe delivery of therapeutic RNAi to patients. Although there are a number of books that cover the subject of RNAi technology, this one is among the first to tackle the important issue of delivery of these molecules in order to silence disease-causing gene expression. It provides a number of protocols for investigators interested in the delivery of therapeutic molecules in vivo. In all chapters, the layout of the protocol is straightforward and based on proven examples that render credibility to these diverse techniques.
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Product Details

  • ISBN-13: 9781617794940
  • Publisher: Springer-Verlag New York, LLC
  • Publication date: 12/1/2011
  • Series: Methods in Molecular Biology Series , #555
  • Edition description: 2009
  • Edition number: 1
  • Pages: 148
  • Product dimensions: 7.65 (w) x 10.27 (h) x 0.40 (d)

Table of Contents

1. Development and Application of a Dual-Purpose Nanoparticle Platform for Delivery and Imaging of siRNA in Tumors
Zdravka Medarova, Mohanraja Kumar, Shu-wing Ng, and Anna Moore

2. Targeted Gene Silencing into Solid Tumors with Electrically Mediated siRNA Delivery
Muriel Golzio, Laurent Mazzolini, Aurélie Paganin-Gioanni, and Justin Teissié

3. Liposomal siRNA for Ovarian Cancer
Lingegowda S. Mangala, Hee Dong Han, Gabriel Lopez-Berestein, and Anil K. Sood

4. Therapeutic Applications of RNAi for Silencing Virus Replication
Ralph A. Tripp and Stephen Mark Tompkins

5. RNAi in the Malaria Vector, Anopheles gambiae
Flaminia Catteruccia and Elena A. Levashina

6. RNAi Using a Chitosan/siRNA Nanoparticle System: in vitro and in vivo Applications
Morten Østergaard Andersen, Kenneth Alan Howard, and Jørgen Kjems

7. Lentiviral and Adeno-Associated Vector-Based Therapy for Motor Neuron Disease through RNAi
Chris Towne and Patrick Aebischer

8. Studying Autoimmunity by in vivo RNA Interference
Stephan Kissler

9. Transcriptional Gene Silencing Using Small RNAs
Daniel H. Kim and John J. Rossi

10. Alternative Splicing as a Therapeutic Target for Human Diseases
Kenneth J. Dery, Veronica Gusti, Shikha Gaur, John E. Shively, Yun Yen, and Rajesh K. Gaur

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