Efforts in gene therapy have grown dramatically in recent years. Basic research as well as clinical activity have made exciting progress and are beg- ning to offer renewed hope that gene therapy may be able to deliver novel approaches for the treatment of inherited as well as such acquired diseases as cardiovascular disease and cancer. With the sequencing of the human genome complete, we now have a comprehensive catalog of genes that further expands the potential role of gene therapy into such new fields as tissue engineering. Central to gene therapy is the process of gene transfer; thus, advances in the technology of gene transfer are at the heart of this field’s progress. Numerous technologies, based on a variety of methods (e.g., viral-mediated, physical/ chemical), have been developed to achieve gene transfer. Some of the earliest methods, such as recombinant retroviruses, are still widely used, have undergone significant improvements, and have given rise to new vectors based on lentiviruses.
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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.
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