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OverviewThe best theories are usually those that explain much in simple terms. There is a certain beauty to such ideas. Physicists in search of a theory of everything, for example, like to admire the aesthetic qualities of their theoretical constructs. The reasoning behind gene therapy is attractive for this very reason. It is simple: if a genetic disease is caused by a dysfunctioning gene, then the disease state can be corrected by giving the patient the normal, functioning gene. Although gene therapy was initially formulated to treat single gene defects, it now holds promise for a wide range of disorders, including cancer, heart disease, and degenerative neurologic disorders. Potentially, it could be a treatment of everything, or at least of many things. Initial attempts at gene therapy involved the genetic modification of cells in culture, which were then transplanted back into the body. This is known as an indirect, or ex vivo, approach. The focus of this book is the simpler, in vivo approach that involves the direct introduction of genes into the body without cell transplantation. The direct approach resembles conventional pharmaceutical delivery methods and promises to be much less expensive than the indirect, ex vivo approaches. There is a certain elegance to its simplicity. Of course, patients care only for alleviation of their disease. They are concerned with risk versus benefit ratios, not with the aesthetics of the theoretical underpinnings of their treatment. Only the results of clinical trials will enable us to judge the clinical value of direct gene therapeutics. However, the tremendous progress reported in this book suggests that this approach has a very promising future. The book is divided into three sections. The first provides a scientific background for the concepts involved in gene therapy that include a history of previous experiments, and the production of mouse genetic models. The basic tenets of expression are explored in one chapter that addresses transcription and another chapter that addresses the post-transcriptional elements of expression. The second section covers the spectacular new methodologies and how these systems work. The methods includes naked DNA, oligonucleotides, calcium phosphate precipitation, polylysine-complexes, adenovirus-polylysine DNA complexes, liposomes, electroporation, and particle bombardment. The third section covers applications for specific organs and diseases. They encompass gene delivery to the CNS skeletal muscle, heart, liver, lung, thyroid and joints. Applications for cardiovascular disease, brain tumors, immunization, arthritis, cystic fibrosis, and Duchenne muscular dystrophy are discussed. Pharmacokinetic considerations are also covered in the chapter by Fred Ledley. Full Product DetailsAuthor: J.F. Crow , Jon A. Wolff , Jon A. WolffPublisher: Birkhauser Boston Inc Imprint: Birkhauser Boston Inc Edition: 1994 ed. Dimensions: Width: 15.50cm , Height: 2.60cm , Length: 23.50cm Weight: 0.790kg ISBN: 9780817636500ISBN 10: 0817636501 Pages: 417 Publication Date: 01 February 1994 Audience: College/higher education , Professional and scholarly , Undergraduate , Postgraduate, Research & Scholarly Format: Hardback Publisher's Status: Out of Print Availability: Out of stock  Table of ContentsI Background.- A History of Gene Transfer and Therapy.- Producing Mouse Genetic Models for Human Diseases.- Post-Transcriptional Considerations of Gene Expression: Translation, MRNA Stability, and Poly(A) Processing.- Promoters, Enhancers, and Inducible Elements for Gene Therapy.- II Methods and Mechanisms.- Possible Mechanisms of DNA Uptake in Skeletal Muscle.- Receptor-Mediated Gene Delivery Employing Adenovirus-Polylysine-DNA Complexes.- Gene Transfer in Mammalian Cells Using Liposomes as Carriers.- In Vivo Gene Therapy via Receptor-Mediated DNA Delivery.- Calcium Phosphate-Mediated DNA Transfection.- Cellular Internalization of Oligodeoxynucleotides.- Gene Transfer via Particle Bombardment: Applications of the Accell Gene Gun.- Electrically-Induced DNA Transfer Into Cells. Electrotransfection In Vivo.- III Applications.- Pharmacokinetic Considerations in the Use of Genes as Pharmaceuticals.- Virus-Mediated Genetic Treatment of Rodent Gliomas.- Gene Therapy for Adenosine Deaminase Deficiency and Malignant Solid Tumors.- Development of Herpes Simplex Virus Vectors for Gene Transfer to the Central Nervous System.- Direct Gene Transfer and Catheter Based Gene Delivery Systems: Applications to Cardiovascular Diseases and Malignancy.- Gene Therapy for Arthritis.- Gene Therapy: The Advent of Adenovirus.- In Vivo Gene Transfer Into the Heart.- Receptor-Mediated Targeted Gene Delivery Using Asialoglycoprotein-Polylysine Conjugates.- Retroviral-Mediated Gene Transfer and Duchenne Muscular Dystrophy.- Keyword Index.ReviewsAuthor InformationTab Content 6Author Website:Countries AvailableAll regions |
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