Article
Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2011
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
Abstract excerpt
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 liver may provide a lifetime source of secreted ARSB. We show that intravascular administration of adeno-associated...
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