AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.

@article{Cunningham2009AAV28mediatedCO,
  title={AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.},
  author={Sharon Cunningham and Afroditi Spinoulas and Kevin H. Carpenter and Bridget Wilcken and Philip W. Kuchel and Ian E. Alexander},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2009},
  volume={17 8},
  pages={1340-6}
}
Ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is associated with severe hyperammonemia accompanied by a high risk of neurological damage and death in patients presenting with the neonatal-onset form. Contemporary therapies, including liver transplantation, remain inadequate with considerable morbidity, justifying vigorous investigation of alternate therapies. Clinical evidence suggests that as little as 3% normal enzyme activity is sufficient to ameliorate… CONTINUE READING
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