In vivo correction with recombinant adenovirus of 4-hydroxyphenylpyruvic acid dioxygenase deficiencies in strain III mice.

@article{Kubo1997InVC,
  title={In vivo correction with recombinant adenovirus of 4-hydroxyphenylpyruvic acid dioxygenase deficiencies in strain III mice.},
  author={Shuji Kubo and Kohji Kiwaki and Hisataka Awata and Hideki Katoh and Yumi Kanegae and Izumu Saito and T Yamamoto and Jun-ichi Miyazaki and Ichiro Matsuda and Fumio Endo},
  journal={Human gene therapy},
  year={1997},
  volume={8 1},
  pages={
          65-71
        }
}
Tyrosinemia type 3, caused by a genetic deficiency of 4-hydroxyphenylpyruvic acid dioxygenase (HPD) in tyrosine catabolism, is characterized by convulsion, ataxia, and mental retardation. The III mouse is a model of tyrosinemia type 3. HPD activity and protein are defective in the liver and its blood tyrosine levels are elevated, the range being between 1,100 and 1,656 microM. We constructed a recombinant adenoviral vector bearing the human HPD cDNA (AdexCAGhHPD), which is expressed under the… 

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