Article
Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pahenu2 mice
The Journal of Gene Medicine
(2003)
Abstract
Treatment of many inherited liver enzyme deficiencies requires the removal of toxic intermediate metabolites from the blood of affected individuals. We propose that circulating toxins can be adequately cleared and disease phenotype influenced by enzyme expressed in tissues other than the liver, such as bone marrow. Our specific hypothesis was that phenylalanine hydroxylase (PAH) expressed in bone marrow would lower blood phenylalanine levels in hyperphenylalaninemic Pahenu2 mice, a model of human phenylketonuria (PKU).
Keywords
- Hyperphenylalaninemia
Disciplines
Publication Date
June, 2003
DOI
10.1002/jgm.432
Publisher Statement
Copyright © 1999 - 2017 John Wiley & Sons, Inc.
Citation Information
Cary O. Harding, Mark W. Neff, Kelly Jones, Krzysztof Wild, et al.. "Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pahenu2 mice" The Journal of Gene Medicine Vol. 5 Iss. 11 (2003) p. 984 - 993 Available at: http://works.bepress.com/mark-neff/7/