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Keywords: enzyme-replacement therapy
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Biochem Soc Trans (2010) 38 (6): 1484–1488.
Published: 24 November 2010
...-like growth factor) II receptor provides an avenue for endocytosis, trafficking and lysosomal processing of extracellularly delivered enzyme. The present review discusses therapeutic utilization of cross-correction by enzyme-replacement therapy, gene therapy and stem cell therapy for the NCLs, along...
Biochem Soc Trans (2010) 38 (6): 1465–1468.
Published: 24 November 2010
... been remarkable progress and there are now licensed treatments for seven of these diseases. In some cases, a choice of agents is available. For selected enzyme-deficiency disordes, ERT (enzyme-replacement therapy) has proved to be highly effective. In other cases, ERT has been less impressive...