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单词 enzyme replacement therapy
释义

enzyme replacement therapy


enzyme replacement therapy

n. The administration of usually synthetic enzymes to limit or control the effects of a disease involving a specific enzyme deficiency, especially the use of pancreatic enzymes in the treatment of cystic fibrosis or other disorders of the pancreas, or the replacement of any of various enzymes implicated in disorders of lysosome storage.

enzyme replacement therapy


enzyme replacement therapy

A 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 therapy

Administration of enzymes to patients with congenital or acquired enzyme deficiency diseases, e.g., oral pancreatic enzymes to patients with chronic pancreatitis, or infused enzymes to patients with genetic disorders in which a single enzyme is lacking, such as galactosidase to patients with Fabry disease.

enzyme replacement therapy

Treatment 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
See ERT
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更新时间:2024/12/24 7:33:06