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Home > Science & Nature > Life Sciences > Life Sciences - Genetics & Genomics > Concepts in Genetic Medicine

Concepts in Genetic Medicine

 
 
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Concepts in Genetic Medicine
A collection of reviews that highlight the most important issues in the development, manufacture, and commercialization of novel genetic medicines

While the field of gene therapy is still a relatively young discipline, it is rapidly evolving and is characterized by impressive scientific and medical innovation. In Concepts in Genetic Medicine, readers are offered a collection of uniformly 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.

Beginning with an introduction to the genetic basis of disease and a discussion of specific diseases, their etiology, and promising therapeutic treatments, the book goes on to cover vector design, genetic payloads and disease targets, vaccine vector systems, manufacture, assays, cell systems and target issues, preclinical testing, regulatory considerations, clinical evaluation, safety concerns, and commercialization.

Complete with uniformly designed illustrations and a supplementary Web site with a PowerPoint presentation for educators, this is a practical guide for physicians, scientists, allied health workers, and patient advocacy professionals attempting to develop genetic medicines for translation into the clinic.


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Title of ebook: Concepts in Genetic Medicine
ISBN: 9780470184578
parent-ISBN: 9780471703204
Publisher: John Wiley & Sons, Inc.
Internet download file size: 4178 kb
Pages: 377
Published: 03-2008
Released online for download: 03-31-2008
Editor: Pratt, Shannon P.
Editor: Carter, Barrie
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Chapter One

The Evolution of Human Gene Therapy: A Journey from Excessive Hype to Excessive Diffidence to Reality

THEODORE FRIEDMANN Center for Molecular Genetics, School of Medicine, University of California at San Diego, La Jolla, California

Unlike Athena, who emerged from the brow of Zeus fully armed and ready for her godly duties, advances in biomedicine are not born fully formed and mature. Virtually all of the therapies and preventive methods that we take for granted-cancer chemotherapy, immunization techniques, tissue transplantation, management of cardiovascular disease, and treatment of diabetes and many other metabolic and degenerative diseases-have required decades of development and incremental advance from initial concept and early proof of concept to truly effective and widely applicable clinical application. They are all still imperfect but are evolving rapidly, and their practitioners are learning from false starts, detours, reversals, and missteps. In most cases, scientists, the public, policymakers, and the media understand and accept what is often a discouragingly slow pace of advance in a difficult new ... read full excerpt from Concepts in Genetic Medicine ebook



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