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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

โœ Scribed by Pike-Overzet, K; Rodijk, M; Ng, Y-Y; Baert, M R M; Lagresle-Peyrou, C; Schambach, A; Zhang, F; Hoeben, R C; Hacein-Bey-Abina, S; Lankester, A C


Book ID
109887434
Publisher
Nature Publishing Group
Year
2011
Tongue
English
Weight
944 KB
Volume
25
Category
Article
ISSN
0887-6924

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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