Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.

@article{Harrington2016NeutralizingAA,
  title={Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.},
  author={Elizabeth A Harrington and Jennifer L Sloan and Irini Manoli and Randy J. Chandler and Mark Schneider and Peter J McGuire and Roberto Calcedo and James Michael Wilson and Charles P. Venditti},
  journal={Human gene therapy},
  year={2016},
  volume={27 5},
  pages={345-53}
}
Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many affected individuals, especially those with mut(0) MMA, has led centers to use elective liver transplantation as a treatment for these patients. We have previously demonstrated the efficacy of systemic… CONTINUE READING
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