Concepts in genetic medicine - Boro Dropulic

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.