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The field of genetics is rapidly evolving, and new medical breakthroughs are occurring as a result of advances in our knowledge of genetics. Advances in Genetics continually publishes important reviews of the broadest interest to geneticists and their colleagues in affiliated disciplines. - Includes methods for testing with ethical, legal, and social implications - Critically analyzes future directions - Written and edited by recognized leaders in the field

This volume of Methods in Enzymology looks at Gene Transfer Vectors for Clinical Application. The chapters provide an invaluable resource for academics, researchers and students alike. With an international board of authors, this volume covers such topics as General principles of retrovirus vector design, Chronic granulomatous disease (CGD), Gene therapy for blindness, and Retrovirus genetic strategy and vector design. - Chapters provide an invaluable resource for academics, researchers and students alike - International board of authors - This volume covers such topics as general principles of retrovirus vector design, chronic granulomatous disease (CGD), gene therapy for blindness, and retrovirus genetic strategy and vector design

This book provides a 2012 reference for state-of-the-art gene transfer technology and the different aspects of its clinical translation with a focus on European-based initiatives. As examples of successful outcomes, recent clinical trials are presented together with Ethical, Safety and Regulatory issues, which are discussed. The broad range of various technologies is covered whether addressing direct in vivo gene transfer like with AAV, Adeno or non-viral vectors or ex-vivo genetically engineered cells including induced pluripotent stem cells (iPS) with integrating vectors such as retrovirus, lentivirus or transposon-derived systems. The critical path to clinical implementation is covered in the second part describing currently available tools - such as molecular imaging, ex-vivo organ cultures and high-throughput technologies used for evaluation of criteria towards a go-or-no go decision to move to the clinic; in addition, utmost salient biosafety and immunotoxicology aspects are discussed. This book is ideal for postgraduates, undergraduates, scientists, clinicians, regulators and patients'advocacy groups looking for states-of-the-art information as well as emerging prospects - including gene targeting and homologous recombination - in gene transfer intended for clinical translation.

This book delivers a collection of organized and succinct reviews in the field of therapeutic genetic medicine presented by a carefully selected group of top experts. Each chapter focuses on a single, current topic and explains issues in the development of genetic treatments, critical challenges and strategies for implementing them, and future directions in research and translational applications. The book is complemented with uniformly designed illustrations and a supplementary Web site with a PowerPoint presentation for educators.

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.

Presents up-to-date summaries of recently completed and ongoing clinical trials.With writings from more than 35 internationally renowned experts, Gene Therapy in Lung Disease unlocks the biological mysteries ofinfection immunity cytokine behavior fibrosis and illustrates the use of gene the

Significant advances in gene transfer technology and the completion of sequencing the human genome offer much renewed hope that gene therapy will provide novel approaches to the treatment of inherited and acquired diseases. In this fully updated and revised 2nd edition of the warmly received Gene Therapy Protocols, leading experts from academic and industrial laboratories around the world detail their most effective viral and nonviral methods of gene transfer, as well as discuss their applications in different organ systems. The methods range from those in which new molecular conjugates show great promise for targeting targeting gene transfer and regulating transgene expression, to those used in such exciting applications as the delivery of therapeutic proteins, vaccination, and tissue engineering. In addition, there are completely revised methods based on retroviruses and adenoviruses, as well as new and promising methods based on lentiviruses and adeno-associated viruses. The quantitative aspects of gene therapy are strongly emphasized, and the book reflects the many significant recent improvements in gene transfer efficiency, gene regulation, and vector production. Up-to-date and highly practical, Gene Therapy Protocols, 2nd Edition, offers a rich compilation of the revolutionary advances that have recently occurred in gene transfer technology, with each article providing proven step-by-step laboratory procedures that enable successful therapeutic application. --

Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements. First, the affected gene must be cloned, its se­ quence determined and its regulation adequately characterized. Second, a suitable vector for the delivery of a good copy of the affected gene must be available. For a vector to be of use several attributes are highly desirable: these include ability to carry the intact gene (although this may be either the genomic or the cDNA form) in a stable form, ability to introduce the gene into the desired cell type, ability to express the introduced gene in an appropriately regulated manner for an extended period of time, and a lack of toxicity for the recipient. Also of concern is the frequency of cell transformation and, in some cases, the ability to introduce the gene into nondividing stem cells. Sev­ eral animal viruses have been tested as potential vectors, but none has proven to have all the desired properties described above. For example, retroviruses are difficult to propagate in sufficient titers, do not integrate into nondividing cells, and are of concern because of their oncogenic properties in some hosts and because they integrate at many sites in the genome and, thus, are potentially insertional mutagens. Additionally, genes introduced by retroviral vectors are frequently expressed for relatively short periods of time. A second virus used as a vector in model systems has been adenovirus (Ad).