Viral Vectors for Gene Therapy: Methods and Protocols

ISBN-13: 9781617373084 / Angielski / Miękka / 2010 / 592 str.

Curtis A. Machida

Opis

Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdoctoral training. The Notes section contained in each chapter provides valuable troublesho- ing guides to help develop working protocols for your laboratory. With Viral Vectors for Gene Therapy: Methods and Protocols, it has been my intent to develop a comprehensive collection of modern molecular methods for the construction, development, and use of viral vectors for gene transfer and gene therapy. I would like to thank the many chapter authors for their contributions. They are all experts in various aspects of viral vectors, and I appreciate their efforts and hard work in developing comprehensive chapters. As editor, it has been a privilege to preview the development of Viral Vectors for Gene Therapy: Methods and Protocols, and to acquire insight into the various methodological approaches from the many different contri- tors."

Recenzje

"This text would be very good for someone who can appreciate the many and varied practical aspects of performing a gene therapy study. I would certainly recommend it to anyone I knew working in the gene therapy field as a book to compare and contrast practical approaches and requirements for various viral vectors." - Pharmaceutical Research

Spis treści

Use of the Herpes Simplex Viral Genome to Construct Gene Therapy Vectors Edward A. Burton, Shaohua Huang, William F. Goins, and Joseph C. Glorioso Construction of Multiply Disabled Herpes Simplex Viral Vectors for Gene Delivery to the Nervous System Caroline E. Lilley and Robert S. Coffin Improved HSV-1 Amplicon Packaging System Using ICP27-Deleted, Oversized HSV-1 BAC DNA Yoshinaga Saeki, Xandra O. Breakefield, and E. Antonio Chiocca Herpes Simplex Amplicon Vectors Charles J. Link, Nicholas N. Vahanian, and Suming Wang Strategies to Adapt Adenoviral Vectors for Targeted Delivery Catherine R. O'Riordan, Antonius Song, and Julia Lanciotti Use of Recombinant Adenovirus for Gene Transfer into the Rat Brain: Evaluation of Gene Transfer Efficiency, Toxicity, and Inflammatory and Immune Reactions Andres Hurtado-Lorenzo, Anne David, Clare Thomas, Maria G. Castro, and Pedro R. Lowenstein Generation of Adenovirus Vectors Devoid of All Virus Genes by Recombination Between Inverted Repeats Hartmut Stecher, Cheryl A. Carlson, Dmitry M. Shayakhmetov, and André Lieber Packaging Cell Lines for Generating Replication-Defective and Gutted Adenoviral Vectors Jeffrey S. Chamberlain, Catherine Barjot, and Jeannine Scott Improving the Transcriptional Regulation of Genes Delivered by Adenovirus Vectors Semyon Rubinchik, Jan Woraratanadharm, Jennifer Schepp, and Jian-yun Dong Targeted Integration by Adeno-Associated Virus Matthew D. Weitzman, Samuel M. Young, Jr., Toni Cathomen, and Richard Jude Samulski Development and Optimization of Adeno-Associated Virus Vector Transfer into the Central Nervous System Matthew J. During, Deborah Young, Kristin Baer, Patricia Lawlor, and Matthias Klugmann A Method for Helper Virus-Free Production of Adeno-Associated Virus Vectors Roy F. Collaco and James P.Trempe Novel Tools for Production and Purification of Recombinant Adeno-Associated Viral Vectors Julian D. Harris, Stuart G. Beattie, and J. George Dickson Recombinant Adeno-Associated Viral Vector Types 4 and 5: Preparation and Application for CNS Gene Transfer Beverly L. Davidson and John A. Chiorini Trans-Splicing Vectors Expand the Packaging Limits of Adeno-Associated Virus for Gene Therapy Applications Dongsheng Duan, Yongping Yue, Ziying Yan, and John F. Engelhardt Generation of Retroviral Packaging and Producer Cell Lines for Large-Scale Vector Production with Improved Safety and Titer Thomas W. Dubensky, Jr. and Sybille L. Sauter An Ecdysone-Inducible Expression System for Use with Retroviruses Karen Morse and John Olsen In Vivo Infection of Mice by Replication-Competent MLV-Based Retroviral Vectors Estanislao Bachrach, Mogens Duch, Mireia Pelegrin, Hanna Dreja, Finn Skou Pedersen, and Marc Piechaczyk Development of Simian Retroviral Vectors for Gene Delivery Biao Li and Curtis A. Machida Self-Inactivating Lentiviral Vectors and a Sensitive Cre-loxP Reporter System Lung-Ji Chang and Anne-Kathrin Zaiss Lentiviral Vectors for Gene Transfer to the Central Nervous System: Applications in Lysosomal Storage Disease Animal Models Deborah J. Watson and John H. Wolfe A Highly Efficient Gene Delivery System Derived from Feline Immunodeficiency Virus (FIV) Sybille L. Sauter, Medhi Gasmi, and Thomas W. Dubensky, Jr A Multigene Lentiviral Vector System Based on Differential Splicing Yonghong Zhu and Vicente Planelles Production of Trans-Lentiviral Vector with Predictable Safety John C. Kappes, Xiaoyun Wu, and John K. Wakefield Human Immunodeficiency Virus Type 1-Based Vectors for Gene Delivery to Human Hematopoietic Stem Cells Ali Ramezani and Robert G. Hawley

Blurb

The promise of gene therapy can be realized only if workable vectors can be found to deliver therapeutic genes. In Viral Vectors for Gene Therapy: Methods and Protocols, leading researchers from academia and biotechnology describe proven molecular methods for the construction, development, and use of virus vectors for gene transfer and gene therapy. Offering detailed step-by-step instructions to ensure successful results, these experts detail the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Additional chapters demonstrate the use of virus vectors in the brain and central nervous system. Each protocol includes a discussion of the principles involved, numerous charts and tables, ample references, and notes on possible problems, troubleshooting, and alternative procedures.
Comprehensive and highly practical, Viral Vectors for Gene Therapy: Methods and Protocols provides not only researchers with the basic tools needed to design targeted gene delivery vectors, but also clinicians with an understanding of how to apply viral vectors to the treatment of genetic disorders.