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The sequencing of the human genome has brought human genetics into a new era of study resulting in the generation of an explosive amount of information. Application of genomic, proteomic, and bioinformatics technologies to the study of human genetics has made it possible for human genetic diseases to be studied on an unprecedented scale, both in silico and in the wet lab. This volume provides up-to-date coverage of the broad range of research topics in this fascinating area. In the first part of the book, a whole spectrum of approaches to human genetics research is reviewed for both background and the latest progress. In the second, important topics related to genetic research of various complex human diseases are discussed. The robust content and diverse array of subjects allow the book to serve as both a concise encyclopedia that introduces basic and essential concepts of human genetics and an in-depth review of the current understanding of genetic research in human diseases

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