Prospects for the treatment of phenylketonuria by gene therapy

Gene therapy is being explored as a means of treating a variety of genetic diseases. Because of the central role of the liver in many metabolic pathwa ys, and the relative ease with which foreign genes can be delivered to hepa tocytes, monogenic disorders that result in deficiencies of liver-specific proteins will be among the first targets of somatic gene therapy. Phenylket onuria represents an ideal model system in which to validate many of the pr inciples of somatic gene therapy. This article will review previous efforts in the development of gene therapy for phenylketonuria using recombinant v iral vectors, and provide an update of the recent progress that has been ma de in the development of the viral vector systems that may prove useful in these efforts. (C) 1999 Wiley-Liss, Inc.