The Pharmaceutical Journal
Vol 269 No 7206 p72
13 July 2002

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Gene therapy

'Gene therapy: the use of DNA as a drug', edited by Gavin Brooks. Pp xix+328. Price £29.95. London: Pharmaceutical Press; 2002. ISBN 0 85369 455 9

The complete sequencing and ongoing efforts to annotate the human genome will ultimately yield the so-called "periodic table of life" whereby the sequence and function of every single gene in the human body will be known. Thus, the concept of treating diseases that result from genetic abnormalities by the addition or replacement of specific genes holds great promise for future health care. However, over 300 clinical studies spanning over more than a decade have led to limited success for gene therapy; a fact that has surprised many of the scientists involved in this area. This excellent book, edited by Gavin Brooks, provides a summary of the pros and cons of gene therapy approaches for treating cancer, cardiovascular diseases, AIDS and other infectious diseases, rheumatoid arthritis and neurological disorders. There are also valuable chapters focusing on DNA structure and regulation, the problems of gene delivery and an overview of the Human Genome Project.

A particular strength of the book is the simple, easy-to-read introductions to each of the clinical applications that will appeal to undergraduates and newcomers to the field.

The book concludes, rather surprisingly, with a chapter discussing the advantages and disadvantages of tissue and organ xenotransplantation — a subject not normally considered as gene therapy. Though the book tries to argue that transplantation of cells also involves transfer of genetic material, it is clearly a secondary consequence and not a primary purpose. A chapter discussing other genetic therapies such as antisense oligonucleotide, ribozyme and DNAzymes may have been more preferable.

In summary, Dr Brooks has produced a well-edited book that serves as a valuable addition to the literature. I highly recommend it to those interested in understanding the fundamentals of gene therapy and some of the disease states for which it is currently being applied in clinical studies.

Saghir Akhtar