A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

Mucopolysaccharidosis type I (MPS I) is an inherited α-L-iduronidase (IDUA, I) deficiency in which glycosaminoglycan (GAG) accumulation causes progressive multisystem organ dysfunction, neurological impairment, and death. Current MPS I mouse models, based on a NOD/SCID (NS) background, are short-li...

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Main Authors: Daniel C Mendez, Alexander E Stover, Anthony D Rangel, David J Brick, Hubert E Nethercott, Marissa A Torres, Omar Khalid, Andrew MS Wong, Jonathan D Cooper, James V Jester, Edwin S Monuki, Cian McGuire, Steven Q Le, Shih-hsin Kan, Patricia I Dickson, Philip H Schwartz
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116300122

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http://www.sciencedirect.com/science/article/pii/S2329050116300122

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