| http://purl.uniprot.org/citations/26052536 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/26052536 | http://www.w3.org/2000/01/rdf-schema#comment | "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-lived, providing a very narrow window to assess the long-term efficacy of therapeutic interventions. They also develop thymic lymphomas, making the assessment of potential tumorigenicity of human stem cell transplantation problematic. We therefore developed a new MPS I model based on a NOD/SCID/Il2rγ (NSG) background. This model lives longer than 1 year and is tumor-free during that time. NSG MPS I (NSGI) mice exhibit the typical phenotypic features of MPS I including coarsened fur and facial features, reduced/abnormal gait, kyphosis, and corneal clouding. IDUA is undetectable in all tissues examined while GAG levels are dramatically higher in most tissues. NSGI brain shows a significant inflammatory response and prominent gliosis. Neurological MPS I manifestations are evidenced by impaired performance in behavioral tests. Human neural and hematopoietic stem cells were found to readily engraft, with human cells detectable for at least 1 year posttransplantation. This new MPS I model is thus suitable for preclinical testing of novel pluripotent stem cell-based therapy approaches."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.org/dc/terms/identifier | "doi:10.1038/mtm.2014.68"xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Brick D.J."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Torres M.A."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Cooper J.D."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Khalid O."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Wong A.M."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "McGuire C."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Kan S.H."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Dickson P.I."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Monuki E.S."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Schwartz P.H."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Jester J.V."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Le S.Q."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Mendez D.C."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Nethercott H.E."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Rangel A.D."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/author | "Stover A.E."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/date | "2015"xsd:gYear |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/name | "Mol Ther Methods Clin Dev"xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/pages | "14068"xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/title | "A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I."xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://purl.uniprot.org/core/volume | "2"xsd:string |
| http://purl.uniprot.org/citations/26052536 | http://www.w3.org/2004/02/skos/core#exactMatch | http://purl.uniprot.org/pubmed/26052536 |