Thirty protocols by leading researchers describe in detail all the essential molecular methods for working with gene transfer systems, along with the methods for gene transfer to specific tissue types either in vivo or ex vivo. The easily reproducible methods range from those for specific viral and nonviral for both genetic and acquired diseases, to those concerned with gene delivery to particular tissues. Methods for applying specific therapeutic systems, such as ribozymes and tumor supressor genes for the treatment of AIDS and cancer, are also included in this authoritative collection.

This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.

In this book internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors and Design and Characterization of Gene Transfer Vectors. Readers will find a comprehensive resource of current and emerging methods for the production of viral and non-viral gene transfer vectors, as well as detailed protocols for applications in stem cell biology, cancer research and infectious disease.

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.

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 Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, 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 BiologyTM series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols 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.

In Gene Therapy Protocols, leading researchers describe in detail all the essential molecular methods for developing gene transfer systems, along with the methods for introducing genes into specific tissue types either in vivo or ex vivo. These easily reproducible methods range from those for specific viral and nonviral delivery systems, to those concerned with gene delivery to particular tissues. Methods for applying specific therapeutic systems, such as ribozymes and tumor suppressor genes for the treatment of AIDS and cancer, are also included in this authoritative collection. Gene Therapy Protocols is the first major collection of the methods needed for successful gene delivery and subsequent in vivo gene expression, techniques at the center of the many and significant recent advances in the treatment of both genetic and acquired diseases. It will surely become today's indispensable standard reference source for all scientists working to realize the promises of gene therapy.

Cell gene engineering is emerging as a field with outstanding impact, not only in medicine/biology, but also, and perhaps most importantly, in agriculture and in all those food sciences involved in the fight against world hunger. Lentivirus vector-based technologies represent the last frontier in the development of powerful and reliable methods for both in vitro and in vivo gene transfer in eukaryotic animal cells. Although the design of lentivirus vectors is closely reminiscent of those already successfully applied to the construction of oncoretroviral vectors, some unique features, e.g., the ef- ciency in transducing both postmitotic and stem cells, render the use of lentivirus vectors invaluable. It has been a great pleasure to edit Lentivirus Gene Engineering Pro- cols, owing in part to the high level of enthusiasm that the authors dem- strated in contributing to this book. The fact that so many outstanding scientists engaged in lentivirus vector research have provided articles renders it so- thing more than a technical handbook. In addition to detailed descriptions of the most innovative methodologies, the reader may find very informative ov- views concerning both theoretical and practical aspects of the origin and the development of diverse lentivirus vector types. This, in my opinion, rep- sents a unique added value of this volume, which should help our work resist the passage of time, to which books such as this are particularly sensitive.