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Clinical Chemistry 22: 323-326, 1976;
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Clinical Chemistry, Vol 22, 323-326, Copyright © 1976 by American Association for Clinical Chemistry

Use of enzyme-loaded erythrocytes in in-vitro correction of arginase- deficient erythrocytes in familial hyperargininemia

K Adriaenssens, D Karcher, A Lowenthal and HG Terheggen

The capacity of arginase-deficient erythrocytes of patients with familial hyperargininemia to produce urea and to catabolize arginine can be increased in vitro by introducing human liver arginase into their erythrocytes. The results of this study on a specific human model show that it is possible to change the metabolic function of a genetically defective erythrocyte by incorporating exogenous human enzyme. The in vivo application of enzyme-loaded erythrocytes for enzyme replacement therapy of inborn metabolic errors in humans must await in vivo studies on animal models.





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Copyright © 1976 by the American Association for Clinical Chemistry.