Long-term Amelioration of Feline Mucopolysaccharidosis VI After AAV-mediated Liver Gene Transfer

Mucopolysaccharidosis VI (MPS VI) is caused by deficient arylsulfatase B (ARSB) activity resulting in lysosomal storage of glycosaminoglycans (GAGs). MPS VI is characterized by dysostosis multiplex, organomegaly, corneal clouding, and heart valve thickening. Gene transfer to a factory organ like liv...

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Hlavní autoři: Cotugno, Gabriella, Annunziata, Patrizia, Tessitore, Alessandra, O'Malley, Thomas, Capalbo, Anita, Faella, Armida, Bartolomeo, Rosa, O'Donnell, Patricia, Wang, Ping, Russo, Fabio, Sleeper, Meg M, Knox, Van W, Fernandez, Steven, Levanduski, Leah, Hopwood, John, De Leonibus, Elvira, Haskins, Mark, Auricchio, Alberto
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2011
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3048181/
https://ncbi.nlm.nih.gov/pubmed/21119624
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.257
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