Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α--Iduronidase in Mice With Mucopolysaccharidosis Type I

Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease characterized by mutations to the α--iduronidase (IDUA) gene resulting in inactivation of the IDUA enzyme. The loss of IDUA protein results in the progressive accumulation of glycosaminoglycans within the lysosomes resulting in seve...

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Autors principals: Osborn, Mark J, McElmurry, Ron T, Lees, Christopher J, DeFeo, Anthony P, Chen, Zhi-Ying, Kay, Mark A, Naldini, Luigi, Freeman, Gordon, Tolar, Jakub, Blazar, Bruce R
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2011
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3048178/
https://ncbi.nlm.nih.gov/pubmed/21081900
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.249
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