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Articles, 1 records found
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7 p, 651.7 KB Toward a gene therapy for neurological and somatic MPSIIIA / Haurigot Mendonça, Virginia (Universitat Autònoma de Barcelona. Departament de Bioquímica i de Biologia Molecular) ; Bosch i Tubert, Fàtima (Universitat Autònoma de Barcelona. Departament de Bioquímica i de Biologia Molecular)
Mucopolysaccharidosis Type IIIA (MPSIIIA) represents an unmet medical need. MPSIIIA shares with many other lysosomal storage disorders (LSD) the characteristic of being a severe neurodegenerative disease accompanied by mild somatic involvement. [...]
2013 - 10.4161/rdis.27209
Rare Diseases, Vol. 1 Núm. 1 (desembre 2013) , p. e27209  

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