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Recombinant adeno-associated virus mediated gene transfer in a mouse model for homocystinuria

Park ES, Oh HJ, Kruger WD, Jung SC, Lee JS

  • KMID: 1115943
  • Exp Mol Med.
  • 2006 Dec;38(6):652-661.
Homocystinuria is a metabolic disorder caused by a deficiency of cystathionine b-synthase (CBS). The major clinical symptoms of this disease are mental retardation, lens dislocation, vascular disease with life-threatening thromboembolisms,...
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