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Prospects for the treatment of phenylketonuria by gene therapy

โœ Scribed by Eisensmith, Randy C. ;Kuzmin, Alexei I. ;Krougliak, Valeri A.

Publisher
John Wiley and Sons
Year
1999
Tongue
English
Weight
92 KB
Volume
5
Category
Article
ISSN
1080-4013

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โœฆ Synopsis

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 pathways, and the relative ease with which foreign genes can be delivered to hepatocytes, monogenic disorders that result in deficiencies of liver-specific proteins will be among the first targets of somatic gene therapy. Phenylketonuria represents an ideal model system in which to validate many of the principles of somatic gene therapy. This article will review previous efforts in the development of gene therapy for phenylketonuria using recombinant viral vectors, and provide an update of the recent progress that has been made in the development of the viral vector systems that may prove useful in these efforts.

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