Gene Therapy: A Primer for Physicians, Second EditionKenneth W. Culver . New York: Mary Ann Liebert, Inc., 1996. 198 pp. $59. ISBN 0-913113-25-5

Extract

A lot of hype and hope surrounds the possible clinical use of gene therapy as a means of treating various genetic as well as other medical diseases, including cancer. The primer, written by Kenneth W. Culver, goes a long way to explain, in simple terms, the present state of this interesting therapeutic modality. It does not, however, tell the reader whether gene therapy works, but the reader is left with no uncertain doubt that gene therapy is here to stay and that it has a certain future in the therapy for patients with a wide array of problems.

Dr. Culver has presented in a logical stepwise fashion the progress of gene therapy from its historic beginnings, through the initial marking studies, and finally now onto the early results of the first therapeutic program involving children with ADA deficiency. Gene therapy requires that the gene of interest be identified and synthesized and that an efficient means of transferring the gene into the cells of interest be available. The first three chapters of Dr. Culver's book very nicely take the reader through the progress that has been made in the field of molecular biology in terms of the identification of genes and the development of technologies aimed at transferring genes into cells. The various methods of gene transfer—namely, direct injection of naked DNA, receptor-mediated transfer,and the use of viruses to transfer genetic material—are discussed in some great detail so that the reader can clearly determine the advantages and disadvantages of each technique. Retroviral gene transfer is noted to be the most efficient means of transferring genes, while the disadvantages of the limitation of the size of the gene that can be transferred and the possibilities of insertional mutagenesis due to the random insertion of the inserted gene are amply discussed. The problem of creating a productive recombinant retroviral infection is not adequately discussed nor are the methods of avoiding this problem addressed (the development of newer generations of retroviral vectors in which this possible side effect is significantly reduced or even potentially completely removed).