Gene therapy: A handbook for physicians

✍ Scribed by Seashore, Margretta R.

• Publisher: John Wiley and Sons
• Year: 1996
• Tongue: English
• Weight: 2 KB
• Volume: 61
• Category: Article
• ISSN: 0148-7299
• DOI: 10.1002/(sici)1096-8628(19960122)61:3<295::aid-ajmg18>3.0.co;2-o

✦ Synopsis

The topic of gene therapy is never far from the clinical geneticist's consciousness these days. Here is a book, written by a leader in this rising field, which sets forth the history of the interest in gene therapy, the role of knowledge about the genome in the development of new medical treatments, and an assessment of the expectations for the applicability of this promising new tool. The author hopes to educate physicians and thus, also, benefit patients. I t appears that the general physician is the intended audience, but the geneticist who has not been working in gene therapy will find useful information as well.

For the nongeneticist reader, the first few chapters provide a very brief introduction to modern molecular genetics. This is followed by a chapter on the methodology of gene transfer and a description of the adenosine deaminase deficiency (ADA) gene therapy experiment. Applications to single disorders, treatment of cancer, and ethical considerations follow. Five appendices offer a selection of resources, including a list of protocols approved by the NIH Recombinant DNA Advisory Committee (RAC), a glossary, an index of health disorders and their chromosome location, and a discussion of points to consider in the development of gene therapy protocols. References are included a t the end of each chapter.

Most of the material reviewed in the first two background chapters is well-known to geneticists. However, the information is clearly covered and accessible, but not too oversimplified. It assumes a good knowledge of medicine and basic science. The chapter titled "Identification of Genes in the Human Genome" offers only the most general of overviews, with no detail. Nor do the references suggest any rigorous, up-to-date reading material. In contrast, the chapter that addresses methods for gene transfer offers more detail, and extensive references. Diagrams are simple and easy to understand. Oddly, liposomal gene transfer is discussed much later, instead of in that chapter.

Culver spends a whole interesting and engaging chapter describing the ADA gene therapy experiment. Along with information about ADA, both clinical and