Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer.

Mucopolysaccharidosis type IIIB (MPS IIIB; or Sanfilippo syndrome type B) is a lysosomal disease, due to glycosaminoglycan storage caused by mutations on the alpha-N-acetylglucosaminidase (NAGLU) gene. The disease is characterized by neurological dysfunction but relatively mild somatic manifestation...

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Enregistré dans:
Détails bibliographiques
Auteurs principaux: Villani, Guglielmo R D, Follenzi, Antonia, Vanacore, Borghina, Di Domenico, Carmela, Naldini, Luigi, Di Natale, Paola
Format: Artigo
Langue:Inglês
Publié: 2002
Sujets:
Research Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC1222624/
https://ncbi.nlm.nih.gov/pubmed/12049639
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1042/BJ20011872
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