Book Description
STEM CELL BIOLOGY AND GENE THERAPY

Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman

Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide.

Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies.

This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including:
* Fundamental regulatory mechanisms that operate in stem cells
* Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy
* Delivery systems for therapeutic genes, including an overview of the most promising vectors
* Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more

Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine. ... Read more

Book Description

In Gene Therapy Protocols, Volumes 1 & 2, internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors (Volume 1) and Design and Characterization of Gene Transfer Vectors (Volume 2). The field of gene therapy has undergone remarkable advances, promising to impact human healthcare significantly in the twenty-first century. Todays technologies can deliver genetic material safely and effectively to cells to slow or halt the progression of disease, and to help repair or regenerate damaged or lost tissues. In this first volume of Gene Therapy Protocols: Production and In Vivo Applications 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 critical applications in stem cell biology, cancer, diabetes, HIV and tissue engineering. This first volume of the new and completely revised third edition of Gene Therapy Protocols will prove a necessary tool for graduate students and postdoctoral fellows and invaluable to basic and clinical researchers in both industry and academia.

Book Description
The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer.

Key Features
* Provides in-depth description of targeted systems and treatment strategies
* Explains the underlying cancer biology necessary for understanding a given therapeutic approach
* Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses
* Presents translational focus with emphasis on requirements for clinical implementation
* Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference ... Read more

Book Description
Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far than gene therapy.This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials.

Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject.Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience.Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis.

* Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells
* Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis
* Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use ... Read more

Book Description
Wolfgang Walther and Ulrike Stein survey the rapidly evolving field of cancer gene therapy and provide a broad array of leading-edge protocols for the delivery of therapeutic genes into tumors. Described in step-by-step fashion and enriched with each author's own practical tips, these readily reproducible methods are currently being widely applied in cancer gene therapy investigations, including immunotherapy and tumor vaccination, suicide gene therapy, antioncogene therapy, and antisense and ribozyme gene therapy. Representative strategies are provided for gene targeting and for viral or nonviral gene delivery in cancer therapy, as well as a significant number of clinical protocols for the development of novel cancer gene therapies. Gene Therapy of Cancer offers basic and clinical researchers a broad ranging overview and collection of the most recent advances in gene transfer techniques. Written by leading internationalauthorities, the methods in this volume constitute today's most valuable tools for the study of cancer gene therapy in both the laboratory and clinical trials. ... Read more

Book Description

Integrates critical reviews of the current strategies of gene transfer in cancer, discusses the technologies of gene therapy, and provides the necessary background to interpret the data-offering an objective survey of the advantages and limitations of this therapeutic field to newcomers and established investigators alike.

Book Description
Yale University, New Haven, Connecticut.Clinically-oriented research for surgeons. In vivo and ex vivo therapy in gastrointestinal disease, vascular disease, and cancer. Halftone illustrations. ... Read more

Book Description
This book brings together all major work achieved during thelast decade in the exciting and promising field of cancer genetherapy, focusing on the achievements and definition of future goals.Providing a picture of the vectors available to carry genes in humantumours, it describes the available genes with different mechanisms ofaction as well as different systems that could be used for targetingtumour cells in humans. A comprehensive review of the importantpreclinical and clinical studies in cancer gene therapy, CancerGene Therapy should be of interest to experts as well as tonewcomers to the field. ... Read more

Book Description
In recent years fundamental progress has been made in understanding the molecular mechanism of the pathogenesis of diseases in different areas of medicine. Additionally, the development of a multitude of gene transfer techniques allows to genetically manipulate almost all cells of the body. To use genes instead of their products for therapy is an entirely new concept in medicine, commonly termed "gene therapy". Gene therapy is still in its infancy. The task is enormous because most targets of gene therapy are diseases for which no curative therapy exists. Seeing the large number of attractive gene therapy approach, there is little doubt that it will find a firm place in the clinic. The difficulty in delivering genes to the right place and effectively controlling their expression are outlined in the first section of this book, which considers both viral and physical methods of gene delivery. The second part describes gene therapy of single gene defects, like cystic fibrosis and familial hypercholesterolemia. After a chapter of gene marking part four covers important aspects of gene therapy of cancer. ... Read more

Product Description
Stem cells provide for life-long cell replacement in tissues and organs, and have inherent homing abilities that are critical in therapeutic applications. Stem cells are also the driving force of cancer where genetic/epigenetic alterations culminate in tumorigenesis either in tissue stem cells or in some of their derivatives. As a rare subset of the tumor, cancer stem cells are the only drive of tumor initiation/propagation. Autologous and cancer stem cells are thus the key targets of 1) long-term and transient-regenerative/epigenetic gene therapy and 2) of recurrence-free anticancer therapy, respectively. While cancer stem cell gene therapy still needs time to accomplish, autologous stem cells have been instrumental in the first unequivocal successes for gene therapy whereby ex vivo retrovirally corrected hematopoietic stem cells have been returned to the patients.

This timely book presents 1) the aforementioned stem cell gene therapy achievements that rely on random-integration of therapeutic transgenes into host chromosomes and 2) emerging experimental approaches aimed at eliminating random-integration oncogenic hazards through site-specific integration or gene targeting. Breakthrough endonuclease-boosted gene targeting for gene correction (inherited diseases) or targeted integration of therapeutic transgenes (other disorders) culminating in an efficiency compatible with clinical trials is one of the highlights of the book. Highlights also include the pioneering transplantation of adult pluripotent stem cells as a substitute for tissue-specific stem cells, thereby pinpointing the invaluable potential for stem cell gene therapy applications of autologous cells able to contribute to all three germ layers. Stem cell gene therapy is thus discussed in terms of 1) magnifying stem cell therapeutic homing through transient regenerative gene therapy and 2) of tackling most pathologies (including mitochondrial DNA diseases and ageing disorders) through stem cell repopulation dynamics into appropriate niches (long-term engraftment) and tissues (cell turn-over). Regarding cancer stem cell gene therapy, focus is on both the increasing number of identified tissue-specific cancer stem cells as the ultimate therapeutic targets and on the development of armed stem cells as tumor-homing vectors for targeted anticancer therapy.

Contents: Introduction: Toward a Universal Platform for Autologous Stem Cell Gene Therapy; Long-term Stem Cell Gene Therapy: From Current Clinical Random-Integration Achievements to Stem Cell Gene Targeting/Cybridization: From Pioneering Clinical Retroviral Gene Therapy to Experimental Site-Specific Integrative Gene Therapy; Stem Cell Gene Targeting/Cybridization: Toward Endonuclease-Boosted Gene Repair/Alteration, Custom Site-Specific Integrative Gene Therapy and Transmitochondrial Therapy/Rejuvenation; Adult Pluripotent Stem Cells: Emerging Stem Cell/Gene Therapy Breakthrough; Cancer Stem Cell Gene Therapy: Cancer Stem Cells as Breakthrough Targets of Cancer Gene Therapy; Armed Stem Cells as Tumor-Homing Vectors for Cancer Gene Therapy. ... Read more

Book Description
Viruses in Human Gene Therapy is a comprehensive and up to datereview describing the basic biology and potential application ofviruses as gene transfer reagents. It will be a useful resource forlaboratories involved in any aspect of gene transfer research andwill also provide an excellent introduction for those who wish tobecome acquainted with the field of gene therapy. ... Read more

Book Description

Endovascular embolization therapy has made remarkable progress during the past two decades, driven primarily by improvements in digital imaging, breakthroughs in microcatheter technology and coil design, and the development of new embolic materials. Initially employed in the management of musculoskeletal trauma, embolization therapy now has a wide range of applications. It permits effective non-operative management of major hepatic, splenic, and renal injuries that once posed a tremendous challenge to the trauma surgeon and has also become an integral facet of the modern oncology center.

Book Description
This special report provides an up-to-date overview on the gene therapy industry and contains a wealth of practical information on 105 companies. For each company, information is available on the people to contact, their contact details, a corporate history, the company's technologies and products, financial data, patents held, products in clinical trials, collaborations with other companies and selected publications from the company's scientists.

Almost 200 companies were initially considered for inclusion but just over 100 companies were finally selected as they fulfilled the necessary critieria to be classified as a 'Gene Therapy Company' defined broadly to include cell therapy companies. As part of this project, new companies were discovered that have not been reported in other publications or on the internet.

Dr Jain has spared no effort to ensure that the information is up-to-date and accurate at the time of publishing. In most instances, the information was reviewed by the companies to correct any outdated information or to add any complementary or recent information.

In addition to the detailed descriptions of each company, the data is also presented in summary tabular form so that at a glance the reader can identify collaborations within the gene therapy industry, or determine which companies are pursuing strategies involving viral, non-viral, or other approaches to gene therapy, and which diseases are being targetted. Given that much of the gene therapy research and development is currently being carried out within the industrial sector, it is important for scientists, managers, and investors to have the relevant information at their fingertips. This special report provides that information in one accurate, easy to consult and up-to-date source. ... Read more

Book Description
Gene transfer within humans has been an obstacle until about 10 years ago. At that time, it was found that viral vectors were effective carriers of "healthy genes" into patients' cells. The problem, however, was that viral vectors proved unnecessarily harmful to humans: subjects experienced inflamatory activity and negative immunological responses to the genes. Viral vectors were also unable to meet the needs of the pharmaceutical community: they were not reproducible in large-scale proportions in cost-effective ways.
Thus, research was undertaken to find a safer way to transfer genes to patients without jeopardizing the safety of the patient. And so non-viral vectors were discovered. This volume presents the various non-viral vectors currently under development. Although not methodologically oriented, it will provide the necessary details behind the development of the vectors. This information will prove useful to both researchers and clinicians.

Key Features
* Presents state-of-the art developments of nonviral vectors as tools for modern molecular medicine
* Covers all types of nonviral vectors, from molecular structure to therapeutic application
Provides a comprehensive review of synthetic vectors
* Includes contributions from major investigators and leading experts in the field ... Read more

Book Description
The field of non-viral vector research has rapidly progressed since the publication of the first edition. This new edition is expanded to two separate volumes that contain in-depth discussions of different non-viral approaches, including cationic liposomes and polymers, naked DNA and various physical methods of delivery, as well as a comprehensive coverage of the molecular biological designs of the plasmid DNA for reduced toxicity, prolonged expression and tissue or disease specific genes. New developments such as the toxicity of the non-viral vectors and recent advances in nucleic acid therapeutics are fully covered in these volumes. ... Read more

Book Description

In recent years cell-based technologies have gained significant scientific attention, and have become a matter of intense public debate as well. Namely, (i) tissue engineering, the construction of tissues and whole organs using molecularly-designed resorbable biomaterials to create new tissue de novo with or without transplanting cells; (ii) the potential use of human embryonic stem cells for transplantation and regenerative medicine (with similar potential for adult-derived stem cells); (iii) and gene therapy, in relation with cell transplantation, have taken their places as the most discussed biomedical issues of the day. New findings in biomimetic materials, cell signalling pathways, extracellular matrix receptors and ligands, growth factors, and the human genome project are motivating the developments in these challenging research areas.

Book Description
Current therapies for most human genetic diseases are inadequate. In response to the need for effective treatments, modern molecular genetics is providing tools for an unprecedented new approach to the treatment of diseases; e.g. the direct manipulation of mutant genes or the input on new therapeutic genes. The treatment of human disease by gene transfer has now moved from the theoretical to the practical realm. With the initiation of clinical trials involving somatic gene therapy in different countries, a critical assessment of the different aspects involved with this new technique is necessary. This volume provides an overview on all these interdisciplinary aspects by some well known experts all over the world. ... Read more