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Toward a gene therapy for neurological and somatic MPSIIIA

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. Thus, the main target organ for the development of new...

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Detalles Bibliográficos
Autores principales:	Haurigot, Virginia, Bosch, Fatima
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Landes Bioscience 2013
Materias:	Addendum
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3927492/ https://www.ncbi.nlm.nih.gov/pubmed/25003015 http://dx.doi.org/10.4161/rdis.27209

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