doi: 10.1126/science.181.4101.753.
Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts
- PMID: 4269326
- DOI: 10.1126/science.181.4101.753
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Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts
Science.
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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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