单词 | enzyme replacement therapy |
释义 | enzyme replacement therapyenzyme replacement therapyenzyme replacement therapyenzyme replacement therapyA generic term for therapeutic administration of a congenitally defective or absent enzyme, which may be adminstered:• Directly, by coupling the enzyme to a carrier molecule or by organ transplantation; or • Indirectly, by introducing the gene into the recipient. enzyme replacement therapyenzyme replacement therapyTreatment of genetic diseases in which the disorder is the result of biochemical dysfunction resulting from the absence of a particular enzyme. Enzymes can now be made by recombinant DNA technology and have to be given by intravenous injection. The method has been used successfully to treat Gaucher's disease, a sphingolipidosis caused by the absence of the lysosomal enzyme glucocerebrosidase.See ERT |
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