Viral Vectors for Gene Therapy: Methods and Protocols / Edition 1

by Otto-Wilhelm Merten, Mohamed Al-Rubeai

Overview

The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Prools, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring prools, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular Biology™ series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory prools, and vital tips on troubleshooting and avoiding known pitfalls.

Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Prools provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.

Product Details

ISBN-13:	9781617790942
Publisher:	Springer-Verlag New York, LLC
Publication date:	05/19/2011
Series:	Methods in Molecular Biology , #737
Edition description:	2011
Pages:	450
Product dimensions:	7.20(w) x 10.20(h) x 1.40(d)

Table of Contents

Introduction to Viral Vectors.- Introduction to Gene Therapy: A Clinical Aftermath.- Host Cells and Cell Banking.- Overview of Current Scalable Methods for Purification of Viral Vectors.- Methods to Construct Recombinant Adenovirus Vectors.- Manufacturing of Adenoviruses Vectors: Production and Purification of Helper Dependent Adenovirus.- Manufacturing of Retroviruses.- Lentiviral Vectors.- Adeno-Associated Viruses.- Manufacturing of Adeno-Associated Viruses, for Example: AAV2.- Vector Characterization Methods for Quality Control Testing of Recombinant Adeno-Associated Viruses.- Baculoviruses Mediate Efficient Gene Expression in a Wide Range of Vertebrate Cells.- Herpes Simplex Virus Type 1 (HSV-1)-Derived Recombinant and Amplicon Vectors.- Manufacture of Measles Viruses.- In Vivo Gene Delivery into hCD34+ Cells in a Humanized Mouse Model.- In Vivo Evaluation of Gene Transfer into Mesenchymal Cells (in View of Cartilage Repair).- Ethical Consideration.- Clinical Trials of GMP Products in the Gene Therapy Field.

Editorial Reviews

Reviewer: Omer Iqbal, MD, FACC, FESC (Loyola University Medical Center)
Description: This book details methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products, particularly the most popular viral vector systems of adenovirus, retrovirus and adeno-associated virus.
Purpose: It is intended for scientists interested in this area of gene therapy.
Audience: Some of the chapters describing protocols for the in vivo gene delivery into CD34 and mesenchymal cells as nonexhaustive examples for in vivo gene transfer are intended for practitioners involved in the preclinical and clinical applications of gene therapy vectors. However, students, scientists, and physicians specializing in gene therapy will find this book extremely useful.
Features: Each of the 18 chapters is written by experts in the field of viral vectors and gene therapy. The book details comprehensive methodologies, providing basic principles for the development of gene therapy viral products that are safe and effective. Following an introductory overview of viral vectors and gene therapy, chapters deal with host cells and cell banking, current scalable methods for purification of viral vectors, methods to construct recombinant adenovirus vectors, and manufacturing of adenovirus, adeno-associated, and retrovirus vectors. Other chapters discuss the protocols for the less common viral vector systems of baculovirus, herpes virus, and measles virus. Other chapters deal with protocols for the in vivo gene delivery into CD34 and mesenchymal cells as nonexhaustive examples for in vivo gene transfer. The chapter on characterization and quality control testing of in vivo gene delivery of adeno-associated viral vectors for the treatment of muscular and eye diseases is quite interesting for physician-scientists. A chapter on ethical issues surrounding human gene therapy and the final chapter on how to conduct gene therapy clinical trials are informative and well written.
Assessment: Given the rapid advances in the field of gene therapy, this book is essential, providing useful information on the methodologies that involve virus production, transferring protocols, and evaluation of the efficacy of gene products and other important methodological concepts in gene therapy.

Doody's Review Service

From the reviews:

“Intended for practitioners involved in the preclinical and clinical applications of gene therapy vectors. … students, scientists, and physicians specializing in gene therapy will find this book extremely useful. … Given the rapid advances in the field of gene therapy, this book is essential, providing useful information on the methodologies that involve virus production, transferring prools, and evaluation of the efficacy of gene products and other important methodological concepts in gene therapy.” (Omer Iqbal, Doody’s Review Service, September, 2011)

From the Publisher