Concepts in Genetic Medicine

This book delivers a collection of 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. The book is complemented with uniformly designed illustrations and a supplementary Web site with a PowerPoint presentation for educators.

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.

Doody Review Services

Reviewer:Luis F. Escobar, MD, MS(St. Vincent Hospital and Health Care Center)
Description:This selective compilation of information about technology in the area of gene-based therapies is organized chronologically in terms of the progressive historical evolution of this technology.
Purpose:This book is designed to provide salient examples of the strategies used in gene therapy. It also provides a very selective view of the new technologies in development. It is intended to call attention to the importance of gene therapy as an approach to the treatment of human disease. In addition, it attempts to expose the need for revolutionary products that would contribute to satisfactory treatment of disease. The title of the book may be misleading. It presents technological information appropriate for basic scientists, but little information for clinicians.
Audience:Although the editors do not specify an audience, it is clear from the technological content that the audience is basic scientists involved in the field of gene therapy. The information should be helpful to graduate students in molecular biology with an interest in gene delivery systems.
Features:The entries are well thought out. The well written chapter on nonviral gene delivery systems gives readers a perspective on the value of studies involving delivery of DNA, RNA, and proteins into cells. Dr. Orentas presents a wonderful review of cancer vaccines, and Drs. Riley and June provide a great description of the use of modified T cells for human gene therapy. The book uses a very traditional format, with very few illustrations and long narrative passages. The lack of graphics may present a challenge for readers, particularly those inexperienced in the field.
Assessment:The editors have done an excellent job of recruiting prominent authors to contribute. This is a unique publication, with good reviews of each topic, but it would require periodic updating.