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Science 24 August 1973:
Vol. 181. no. 4101, pp. 753 - 755
DOI: 10.1126/science.181.4101.753
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Articles

Sanfilippo Disease Type B: Enzyme Replacement and Metabolic Correction in Cultured Fibroblasts

J. S. O'Brien 1, A. L. Miller 1, A. W. Loverde 1, and M. L. Veath 1

1 Department of Neurosciences, School of Medicine, University of California, San Diego, La Jolla 92037

agr-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.


THIS ARTICLE HAS BEEN CITED BY OTHER ARTICLES:
Infusion of Normal HL-A Identical Leukocytes in Sanfilippo Disease Type B: Estimate of Infused Cell Survival by Assays of {alpha}-N-Acetylglucosaminidase Activity and Cytogenetic Techniques: Effect on Glycosaminoglycan Excretion in the Urine.
H. W. Moser, J. S. O'Brien, L. Atkins, T. C. Fuller, A. Kliman, S. Janowska, P. S. Russell, C. S. Bartsocas, B. Cosimi, and J. T. Dulaney (1974)
Arch Neurol 31, 329-337
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