Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts

Background Hunter syndrome, mucopolysaccharidosis type II (MPS II), is a X‐linked inherited disorder caused by the deficiency of the enzyme iduronate‐2‐sulfatase (IDS), involved in the lysosomal catabolism of the glycosaminoglycans (GAG) dermatan and heparan sulfate. Such a deficiency leads to the i...

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Veröffentlicht in:	The journal of gene medicine 2005-11, Vol.7 (11), p.1482-1491
Hauptverfasser:	Friso, Adelaide, Tomanin, Rosella, Alba, Sabrina, Gasparotto, Nicoletta, Puicher, Elisabetta Piller, Fusco, Mariella, Hortelano, Gonzalo, Muenzer, Joseph, Marin, Oriano, Zacchello, Franco, Scarpa, Maurizio
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Capsules Cell Transplantation - methods Disease Models, Animal Gene therapy Glycoproteins - genetics Glycoproteins - metabolism Glycosaminoglycans - metabolism Humans Hunter syndrome Kidney - cytology Kidney - metabolism Liver - cytology Liver - metabolism lysosomal enzymes Mice Mice, Inbred C57BL Mice, Knockout microcapsules MPS II mouse model Mucopolysaccharidosis II - genetics Mucopolysaccharidosis II - metabolism Mucopolysaccharidosis II - physiopathology Mucopolysaccharidosis II - therapy Myoblasts - cytology Myoblasts - physiology somatic cell therapy Spleen - cytology Spleen - metabolism
Online-Zugang:	Volltext
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