A Self-inactivating [gamma]-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice

Mucopolysaccharidosis I (MPS I) is a lysosomal storage disease due to deficiency in α-L-iduronidase (IDUA) that results in accumulation of glycosaminoglycans (GAGs) throughout the body, causing numerous clinical defects. Intravenous administration of a γ-retroviral vector (γ-RV) with an intact long...

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Veröffentlicht in:	Molecular therapy 2010-02, Vol.18 (2), p.334
Hauptverfasser:	Metcalf, Jason A, Ma, Xiucui, Linders, Bruce, Wu, Susan, Schambach, Axel, Ohlemiller, Kevin K, Kovacs, Attila, Bigg, Mark, He, Li, Tollefsen, Douglas M, Ponder, Katherine P
Format:	Artikel
Sprache:	eng
Schlagworte:	Deafness Drug dosages Enzymes Gene therapy Growth factors Heparan sulfate Liver Mutagenesis Stem cell transplantation Vectors (Biology)
Online-Zugang:	Volltext
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