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Book and Media Reviews
March 19, 2008

Gene Therapy

JAMA. 2008;299(11):1367. doi:10.1001/jama.299.11.1367-a

This book addresses the important issue of gene therapy in human disease states. Gene therapy, which implies introduction of novel genes and inhibition of disease-causing genes, has been a subject of multiple papers in scientific and lay journals. Naturally, there is considerable interest in this topic among physicians, patients, ethicists, politicians, funding agencies, pharmaceutical companies, and biotechnology corporations.

Evelyn Kelly's Gene Therapy consists of 3 sections: “Scientific Background,” “Ethics and Regulation,” and “References and Resources.” The first section presents the background for gene therapy and various discoveries leading to gene therapy. These chapters are eloquently written, although with some repetition. The reader gets an especially comprehensive idea of the structure of genes and the ingenious work of a host of scientists leading to the discovery of human genes. Early trials of gene therapy in the 1990s are clearly presented. The last 4 chapters in this section are a textbook listing of single-gene recessive disorders, single-gene traits/dominant disorders; X-linked disorders, and multigene traits influenced by the environment. The flow evident in the earlier chapters, however, is lost in the last 4 chapters.

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