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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Detalhes bibliográficos
Main Authors: Anson, Donald S, McIntyre, Chantelle, Thomas, Belinda, Koldej, Rachel, Ranieri, Enzo, Roberts, Ainslie, Clements, Peter R, Dunning, Kylie, Byers, Sharon
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2007
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1783652/
https://ncbi.nlm.nih.gov/pubmed/17227588
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1479-0556-5-1
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