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430. Antibodies to Recombinant Human α-L-Iduronidase Enhance Uptake into Macrophages in Murine Mucopolysaccharidosis Type I
Molecular Therapy
◽
10.1016/s1525-0016(16)33239-7
◽
2016
◽
Vol 24
◽
pp. S170-S171
Author(s):
Valentina Sanghez
◽
Shih-Hsin Kan
◽
Steven Q Le
◽
Don Clarke
◽
Kristen Vondrak
◽
...
Keyword(s):
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
Download Full-text
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Phase I/II clinical trial design for a novel therapy for mucopolysaccharidosis type I with an intravenously administered blood-brain barrier-crossing enzyme (JR-171)
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Phase I
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Trial Design
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Clinical Trial Design
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Brain Barrier
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Mucopolysaccharidosis Type
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Mucopolysaccharidosis Type I
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Novel Therapy
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Sexual behaviour in a murine model of mucopolysaccharidosis type I (MPS I)
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Mucopolysaccharidosis Type
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Mucopolysaccharidosis Type I
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Neuropathology in Mouse Models of Mucopolysaccharidosis Type I, IIIA and IIIB
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Anti-drug antibody response in mucopolysaccharidosis type I patients treated with AGT-181, a brain penetrating human insulin receptor antibody-iduronidase fusion protein
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Fusion Protein
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Insulin Receptor
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Human Insulin
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Mucopolysaccharidosis Type
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Mucopolysaccharidosis Type I
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Evidence of a progressive motor dysfunction in Mucopolysaccharidosis type I mice
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Motor Dysfunction
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69. Gene therapy of mucopolysaccharidosis type I: Intraventricular administration of adeno-associated virus vector transducing the human alpha-l-iduronidase gene in a murine model of the disease
Molecular Genetics and Metabolism
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10.1016/j.ymgme.2007.10.081
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Gene Therapy
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Murine Model
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Virus Vector
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Type I
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Intraventricular Administration
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Mucopolysaccharidosis Type
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Mucopolysaccharidosis Type I
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Adeno Associated Virus
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373. Expression of Human Iduronidase from Sleeping Beauty Engineered Human B Lymphocytes as a Cellular Therapy for Mucopolysaccharidosis Type I
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10.1016/s1525-0016(16)33982-x
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Mucopolysaccharidosis Type
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Mucopolysaccharidosis Type I
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Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I
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Three novel α-L-iduronidase mutations in 10 unrelated Chinese mucopolysaccharidosis type I families
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10.1590/s1415-47572011005000006
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Mucopolysaccharidosis Type
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