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Research literature, 1 records found
Research literature 1 records found  
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76 p, 2.8 MB Intrathecal administration of AAVrh10 coding for β‐glucuronidase corrects biochemical and histological hallmarks of mucopolysaccharidosis type VII mice and improves behavior and survival / Pagès i Pi, Gemma ; Bosch i Merino, Assumpció, dir. (Universitat Autònoma de Barcelona. Departament de Bioquímica i de Biologia Molecular) ; Universitat Autònoma de Barcelona. Departament de Bioquímica i Biologia Molecular
La mucopolisacaridosi tipus VII (MPS VII) és una malaltia monogènica molt rara inclosa en el grup de malalties lisosòmiques. Està causada per la manca d'activitat β-­‐ glucuronidasa, un enzim lisosòmic involucrat en la via de degradació de glicosaminoglicans. [...]
Mucopolysaccharidosis type VII (MPS VII) is an ultrarare monogenic lysosomal storage disease. It is caused by the lack of β-­‐glucuronidase activity, a lysosomal enzyme involved in the degradation pathway of glycosaminoglycans. [...]

[Barcelona] : Universitat Autònoma de Barcelona, 2015
4 documents

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