A Self-inactivating γ-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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Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2009
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839301/
https://ncbi.nlm.nih.gov/pubmed/19844196
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.236
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