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Lentiviral mediated stem cell gene therapy corrects a mouse model of mucopolysaccharidosis type IIIA

โœ Scribed by Brian Bigger; Alex Langford-Smith; Fiona Wilkinson; Kia Langford-Smith; Ana Sergijenko; Ed Wraith; Rob Wynn


Book ID
116989124
Publisher
Elsevier Science
Year
2011
Tongue
English
Weight
58 KB
Volume
102
Category
Article
ISSN
1096-7192

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Correction of mucopolysaccharidosis type
โœ Chantelle McIntyre; Sharon Byers; Donald S. Anson ๐Ÿ“‚ Article ๐Ÿ“… 2010 ๐Ÿ› John Wiley and Sons ๐ŸŒ English โš– 621 KB

## 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