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This is the story of Dr. W. French Anderson who performed the first government-approved human gene transfer in 1990 on a patient stricken with a deadly disease from birth. Anderson has brought humanity into a fourth revolution of medicine, healing disease by genetic engineering.

Anderson is a Tulsa, Oklahoma, native who currently is the director of the Gene Therapy Laboratories at the University of Southern California School of Medicine. ... Read more

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Frrench Anderson, a true rebel with a real cause
Gene therapy may hold the key to a brighter future for those born with a deadly disease.Dr. Anderson has spent nearly 30 years developing procedures to carry out human gene therapy--replacing a defective gene with a healthy gene.In 1990 Anderson performed the first government-approved gene transfer on a 4-year-old Ohio girl.The transfer was a success.This is the story of Anderson's life and how, as a senior at Harvard University, he developed the concept for this type of treatment.The authors take an extremely complicated subject and provide the reader with complete understanding.A must read for all! ... Read more

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Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies; however, it faces a number of unique challenges, including the need to deliver a therapeutic vector to all muscles throughout the body.In Muscle Gene Therapy: Methods and Protocols, expert researchers in the field present a collection of techniques aimed at bridging the translational gap in muscle gene therapy between the prevalent rodent models and vitally important larger animal models.Divided into three sections, this volume examines basic protocols for optimizing the muscle gene expression cassette and for evaluating the therapeutic outcomes, new developments in muscle gene therapy technology such as adeno-associated viral vector (AAV), oligonucleotide-mediated exon-skipping, and novel RNA-based strategies, and step-by-step guidance on muscle gene delivery in swine, ovine, canine, and non-human primates.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, detailed, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls.Authoritative and cutting-edge, Muscle Gene Therapy: Methods and Protocols serves as an invaluable resource for graduate students, post-doctoral fellows, and principle investigators pursuing the crucial advancement of muscle disease gene therapy in the hope of someday curing these debilitating disorders. ... Read more

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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.

Further important indications include uterine fibroids, vascular malformations, visceral aneurysms and GI bleeding. This volume and volume 1 present the current state of the art in this exciting and growing field. High informational content is supplemented by detailed graphics, providing a comprehensive reference work for both experts and novices in the field.

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This book is a collection of preclinical and clinical reports on the application of gene therapy to human disease. The two methods available for delivering therapeutic genes to diseased cells, viral and non-viral vector systems, are detailed and characterized. Several reports describe both approaches currently used in gene therapy: the introduction of a wild-type gene to restore normal gene function in diseased cells, and the use of antisense molecules to hinder abnormal gene expression. Clinical studies detail several different strategies for the treatment of cancer and cardiovascular diseases, using genetic material as therapeutic agents. The regulatory issues governing the use of gene therapy in Europe and the United States are also presented. This book highlights the range of applications and demonstrates the rapid progress being made in the field of gene therapy. ... Read more

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The first unequivocal success for Gene Therapy was reported in April 2000 for X-SCID patients. Pioneering stem cell/gene therapy clinical trials are the focus of this book. Therapy successes such as the X-SCID trial and improved ADA-SCID ones are presented together with pioneering angio/vasculogenic clinical trials mediated either by transient gene therapy or emerging autologous stem cell transplantation. Highlights also include 1) promises of the breakthrough combination of stem cell- and transient gene-therapy, 2) gene therapy trials for neurodegenerative disease on non-human primates where long-term gene therapy might involve brain stem cells, and 3) the first clinical trial with non-invasive monitoring of therapeutic gene expression as a prospective conclusion.

This volume will be of value and interest to researchers in this exciting field. ... Read more

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Tumor Suppressing Viruses, Genes, and Drugs profiles the new generation of cancer treatments now in development. The book examines the innovative new approaches of viral, gene, and signal therapies that promise to replace or enhance conventional methods such as surgery, radiation, and chemotherapy. The timely information presented by this book should be of interest to anyone concerned with advancing cancer treatment beyond current medical practices.

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This report was created for global strategic planners who cannot be content with traditional methods of segmenting world markets. With the advent of a "borderless world", cities become a more important criteria in prioritizing markets, as opposed to regions, continents, or countries. This report covers the top 2000 cities in over 200 countries. It does so by reporting the estimated market size (in terms of latent demand) for each major city of the world. It then ranks these cities and reports them in terms of their size as a percent of the country where they are located, their geographic region (e.g. Africa, Asia, Europe, Middle East, North America, Latin America), and the total world market.

In performing various economic analyses for its clients, I have been occasionally asked to investigate the market potential for various products and services across cities. The purpose of the studies is to understand the density of demand within a country and the extent to which a city might be used as a point of distribution within its region. From an economic perspective, however, a city does not represent a population within rigid geographical boundaries. To an economist or strategic planner, a city represents an area of dominant influence over markets in adjacent areas. This influence varies from one industry to another, but also from one period of time to another.

In what follows, I summarize the economic potential for the world's major cities for "gene therapy" for the year 2009. The goal of this report is to report my findings on the real economic potential, or what an economist calls the latent demand, represented by a city when defined as an area of dominant influence. The reader needs to realize that latent demand may or may not represent real sales. ... Read more

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University Hospital, Frankfurt, Germany. Papers presented at the 30th Hemophilia Symposium, held in Hamburg, Germany, in 1999. Paper topics included HIV infection and epidemiology in hemophilia therapy of Hepatitis C, inhibitors in hemophilia, and pediatric hemostasiology. Softcover. ... Read more

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Massachusetts General Hospital, Boston.Survey of the recent advances in the field. For investigators, graduate and advanced undergraduate students. 53 contributors, 50 U.S. ... Read more

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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

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This econometric study covers the latent demand outlook for gene therapy across the regions of Greater China, including provinces, autonomous regions (Guangxi, Nei Mongol, Ningxia, Xinjiang, Xizang - Tibet), municipalities (Beijing, Chongqing, Shanghai, and Tianjin), special administrative regions (Hong Kong and Macau), and Taiwan (all hereafter referred to as "regions"). Latent demand (in millions of U.S. dollars), or potential industry earnings (P.I.E.) estimates are given across some 1,100 cities in Greater China. For each major city in question, the percent share the city is of the region and of Greater China is reported. Each major city is defined as an area of "economic population", as opposed to the demographic population within a legal geographic boundary. For many cities, the economic population is much larger that the population within the city limits; this is especially true for the cities of the Western regions. For the coastal regions, cities which are close to other major cities or which represent, by themselves, a high percent of the regional population, actual city-level population is closer to the economic population (e.g. in Beijing). Based on this "economic" definition of population, comparative benchmarks allow the reader to quickly gauge a city's marketing and distribution value vis-a-vis others. This exercise is quite useful for persons setting up distribution centers or sales force strategies. Using econometric models which project fundamental economic dynamics within each region and city of influence, latent demand estimates are created for gene therapy. This report does not discuss the specific players in the market serving the latent demand, nor specific details at the product level. The study also does not consider short-term cyclicalities that might affect realized sales. The study, therefore, is strategic in nature, taking an aggregate and long-run view, irrespective of the players or products ... Read more

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This econometric study covers the outlook for gene therapy in the Middle East & Africa. For each year reported, estimates are given for the latent demand, or potential industry earnings (P.I.E.), for the country in question (in millions of U.S. dollars), the percent share the country is of the region and of the globe. These comparative benchmarks allow the reader to quickly gauge a country vis-a-vis others. Using econometric models which project fundamental economic dynamics within each country and across countries, latent demand estimates are created. This report does not discuss the specific players in the market serving the latent demand, nor specific details at the product level. The study also does not consider short-term cyclicalities that might affect realized sales. The study, therefore, is strategic in nature, taking an aggregate and long-run view, irrespective of the players or products involved.

This study does not report actual sales data (which are simply unavailable, in a comparable or consistent manner in virtually all of the countries in the Middle East & Africa). This study gives, however, my estimates for the latent demand, or the P.I.E. for gene therapy in the Middle East & Africa. It also shows how the P.I.E. is divided across the national markets of the Middle East & Africa. For each country, I also show my estimates of how the P.I.E. grows over time (positive or negative growth). In order to make these estimates, a multi-stage methodology was employed that is often taught in courses on international strategic planning at graduate schools of business. ... Read more

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This single-source volume 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. ... Read more

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This econometric study covers the outlook for gene therapy in Europe. For each year reported, estimates are given for the latent demand, or potential industry earnings (P.I.E.), for the country in question (in millions of U.S. dollars), the percent share the country is of the region and of the globe. These comparative benchmarks allow the reader to quickly gauge a country vis-a-vis others. Using econometric models which project fundamental economic dynamics within each country and across countries, latent demand estimates are created. This report does not discuss the specific players in the market serving the latent demand, nor specific details at the product level. The study also does not consider short-term cyclicalities that might affect realized sales. The study, therefore, is strategic in nature, taking an aggregate and long-run view, irrespective of the players or products involved.

This study does not report actual sales data (which are simply unavailable, in a comparable or consistent manner in virtually all of the countries in Europe). This study gives, however, my estimates for the latent demand, or the P.I.E. for gene therapy in Europe. It also shows how the P.I.E. is divided across the national markets of Europe. For each country, I also show my estimates of how the P.I.E. grows over time (positive or negative growth). In order to make these estimates, a multi-stage methodology was employed that is often taught in courses on international strategic planning at graduate schools of business. ... Read more

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This digital document is an article from Pediatric News, published by International Medical News Group on March 1, 2004. The length of the article is 665 words. The page length shown above is based on a typical 300-word page. The article is delivered in HTML format and is available in your Amazon.com Digital Locker immediately after purchase. You can view it with any web browser.

Citation Details
Title: The promise of gene therapy.(Guest Editorial)
Author: Joseph C., III Glorioso
Publication: Pediatric News (Magazine/Journal)
Date: March 1, 2004
Publisher: International Medical News Group
Volume: 38Issue: 3Page: 18(1)

Distributed by Thomson Gale ... Read more

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This econometric study covers the latent demand outlook for gene therapy across the states and cities of the United States. Latent demand (in millions of U.S. dollars), or potential industry earnings (P.I.E.) estimates are given across some 9,500 cities in the United States. For each city in question, the percent share the city is of it's state and of the United States is reported. These comparative benchmarks allow the reader to quickly gauge a city vis-a-vis others. This statistical approach can prove very useful to distribution and/or sales force strategies. Using econometric models which project fundamental economic dynamics within each state and city, latent demand estimates are created for gene therapy. This report does not discuss the specific players in the market serving the latent demand, nor specific details at the product level. The study also does not consider short-term cyclicalities that might affect realized sales. The study, therefore, is strategic in nature, taking an aggregate and long-run view, irrespective of the players or products involved.

This study does not report actual sales data (which are simply unavailable, in a comparable or consistent manner in virtually all of the cities in the United States). This study gives, however, my estimates for the latent demand, or the P.I.E., for gene therapy in the United States. It also shows how the P.I.E. is divided and concentrated across the cities and regional markets of the United States. For each state, I also show my estimates of how the P.I.E. grows over time. In order to make these estimates, a multi-stage methodology was employed that is often taught in courses on strategic planning at graduate schools of business. ... Read more

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Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight disease. Gene therapy is being studied in clinical trials for many different types of cancer and for numerous other diseases. This book offers important research from around the globe dedicated to this dynamic subject. ... Read more