Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy.
about
Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon systemEvidence of a progressive motor dysfunction in Mucopolysaccharidosis type I mice.Decreased performance in IDUA knockout mouse mimic limitations of joint function and locomotion in patients with Hurler syndromeAssessment of dysmyelination with RAFFn MRI: application to murine MPS I.Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler DiseaseLysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Quantitative analysis of α-L-iduronidase expression in immunocompetent mice treated with the Sleeping Beauty transposon systemDirect gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I.AAV Gene Therapy for MPS1-associated Corneal BlindnessCharacterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation.Animal models for metabolic, neuromuscular and ophthalmological rare diseases.Standardization of α-L-iduronidase enzyme assay with Michaelis-Menten kinetics.Liver-Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model.High-dose enzyme replacement therapy in murine Hurler syndromeNeonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I.Substrate Deprivation Therapy to Reduce Glycosaminoglycan Synthesis Improves Aspects of Neurological and Skeletal Pathology in MPS I Mice.Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I.Intracerebroventricular transplantation of human bone marrow-derived multipotent progenitor cells in an immunodeficient mouse model of mucopolysaccharidosis type I (MPS-I).
P2860
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P2860
Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy.
description
2007 nî lūn-bûn
@nan
2007 թուականի Օգոստոսին հրատարակուած գիտական յօդուած
@hyw
2007 թվականի օգոստոսին հրատարակված գիտական հոդված
@hy
2007年の論文
@ja
2007年論文
@yue
2007年論文
@zh-hant
2007年論文
@zh-hk
2007年論文
@zh-mo
2007年論文
@zh-tw
2007年论文
@wuu
name
Characterization of an immunod ...... an stem cell and gene therapy.
@ast
Characterization of an immunod ...... an stem cell and gene therapy.
@en
Characterization of an immunod ...... an stem cell and gene therapy.
@nl
type
label
Characterization of an immunod ...... an stem cell and gene therapy.
@ast
Characterization of an immunod ...... an stem cell and gene therapy.
@en
Characterization of an immunod ...... an stem cell and gene therapy.
@nl
prefLabel
Characterization of an immunod ...... an stem cell and gene therapy.
@ast
Characterization of an immunod ...... an stem cell and gene therapy.
@en
Characterization of an immunod ...... an stem cell and gene therapy.
@nl
P2093
P2860
P1476
Characterization of an immunod ...... an stem cell and gene therapy.
@en
P2093
Indrani Maitra
Leah E Colvin-Wanshura
Matthew S Nelson
Mayra F Garcia-Rivera
Pankaj Gupta
Shaukat A Khan
Tyson B Rogers
Walter C Low
Zhenhong Nan
P2860
P304
P356
10.1016/J.BRAINRESBULL.2007.07.018
P577
2007-08-06T00:00:00Z