Long-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.

@article{Lee2012LongtermSO,
  title={Long-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.},
  author={Eun Kyung Lee and Chuhong Hu and Ragini Bhargava and Nora Rozengurt and David E. Stout and Wayne W. Grody and Stephen D. Cederbaum and Gerald S. Lipshutz},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2012},
  volume={20 10},
  pages={1844-51}
}
Arginase deficiency is characterized by hyperargininemia and infrequent episodes of hyperammonemia. Human patients suffer from neurological impairment with spasticity, loss of ambulation, seizures, and severe mental and growth retardation. In a murine model, onset of the phenotypic abnormality is heralded by weight loss beginning around day 15 with death occurring typically by postnatal day 17 with hyperargininemia and markedly elevated ammonia. The goal of this study was to address the… CONTINUE READING

Citations

Publications citing this paper.
Showing 1-9 of 9 extracted citations

References

Publications referenced by this paper.
Showing 1-10 of 25 references

Similar Papers

Loading similar papers…