Enzyme replacement therapy in Gaucher's and Fabry's disease.
نویسنده
چکیده
Glucocerebrosidase and ceramidetrihexoside-alpha-galactosidase were obtained in a high degree of purity from human placental tissue. The enzymes were infused in Gaucher and Fabry patients, respectively. Following the administration of the proteins to supplement the genetically determined deficiencies, there resulted a specific reduction in the accumulated hlycolipids in the circulation and liver. These results indicate that enzyme replacement may provide hope for the clinical treatment of these disorders.
منابع مشابه
Acid hydrolases in leukocytes and platelets of normal subjects and in patients with Gaucher's and Fabry's disease
Lymphocytes, monocytes, neutrophilic granulocytes and platelets were each separated to greater than 95% purity from six normal subjects, three patients with Gaucher's disease, two heterozygotes for Gaucher's disease, and one patient with Fabry's disease. Activities of the following acid hydrolases were determined: "acid" (pH 4.0) beta-glucosidase, pH 5.0 beta-glucosidase, alpha-galactosidase, a...
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ورودعنوان ژورنال:
- Annals of clinical and laboratory science
دوره 7 3 شماره
صفحات -
تاریخ انتشار 1977