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Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

โœ Scribed by Wolfe, John H.; Sands, Mark S.; Barker, Jane E.; Gwynn, Babette; Rowe, Lucy B.; Vogler, Carole A.; Birkenmeier, Edward H.


Book ID
109782786
Publisher
Nature Publishing Group
Year
1992
Tongue
English
Weight
824 KB
Volume
360
Category
Article
ISSN
0028-0836

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Correction of mucopolysaccharidosis type
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## Abstract ## Background The hallmark of lysosomal storage disorders (LSDs) is microscopically demonstrable lysosomal distension. In mucopolysaccharidosis type IIIA (MPS IIIA), this occurs as a result of an inherited deficiency of the lysosomal hydrolase sulphamidase. Consequently, heparan sulpha