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Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA

BACKGROUND: Mucopolysaccharidosis type IIIA (MPS IIIA) is the most common of the mucopolysaccharidoses. The disease is caused by a deficiency of the lysosomal enzyme sulphamidase and results in the storage of the glycosaminoglycan (GAG), heparan sulphate. MPS IIIA is characterised by widespread stor...

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Detalles Bibliográficos
Autores principales:	Anson, Donald S, McIntyre, Chantelle, Thomas, Belinda, Koldej, Rachel, Ranieri, Enzo, Roberts, Ainslie, Clements, Peter R, Dunning, Kylie, Byers, Sharon
Formato:	Texto
Lenguaje:	English
Publicado:	BioMed Central 2007
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1783652/ https://www.ncbi.nlm.nih.gov/pubmed/17227588 http://dx.doi.org/10.1186/1479-0556-5-1

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