enzyme replacement therapy

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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.
References in periodicals archive ?
But she does not want to risk waiting if she can help it and there is an enzyme replacement treatment, not available on the NHS, but available in America which she wants Brooklyn to have a chance at.

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