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. 1973 Aug 24;181(4101):753-5.
doi: 10.1126/science.181.4101.753.

Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts

J S O'Brien, A L Miller, A W Loverde, M L Veath

Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts

J S O'Brien et al. Science. .

Abstract

alpha-Acetylglucosaminidase, purified from human placent, corrected the defect in mucopolysaccharide degradation when added to culture fibroblasts from patients with Sanfilippo disease type B. A small cellular concentration of enzyme gave a large corrective effect. The half-life of disappearance of enzyme activity was 4 to 7 days.

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