Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 5 of 5 for “"Mucopolysaccharidosis type I"”.
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Molecular therapy for mucopolysaccharidosis Type I
Mucopolysaccharidosis type I (MPS I) is caused by deficiency of the lysosomal hydrolase alpha-L-iduronidase (IDUA). IDUA is a required component of the step-wise degradative pathway responsible for the catabolism of the glycosaminoglycans (GAGs) heparan sulfate and dermatan sulfate. As a result, …
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Alpha-L-iduronidase transduced mesenchymal stem cells as a therapy for the treatment of CNS degeneration in mucopolysaccharidosis type I mice
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive disorder that is characterised by a deficiency in the α-L-iduronidase (IDUA) enzyme, resulting in the accumulation of undegraded heparan sulphate and dermatan sulphate glycosaminoglycans (gags) within the lysosome of nearly every cell. …
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Advances in functional magnetic resonance imaging to facilitate brain research
… an inherited genetic lysosomal disorder named mucopolysaccharidosis type I (MPS I).
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Characterization Of The Skeletal Phenotype In Idua-W392X Knock-In Mice: Bone Metabolism Biomarkers
Mucopolysaccharidosis Type I (MPS I, Hurlers Syndrome) is a lysosomal storage disease caused by a deficiency of alpha-L-iduronidase (IDUA). IDUA catalyzes the degradation of the two glycosaminoglycans (GAGs); heparin sulfate (HS) and demantan sulfate (DS). The accumulation of HS and DS makes MPS I …
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Engineering cell-based micropharmacies for in vivo protein replacement therapy
… micropharmacies for enzyme replacement in Mucopolysaccharidosis type I (MPS I), a lysosomal storage disorder resulting from a deficiency in alpha-L-iduronidase (IDUA). Current treatments, including ERT and hematopoietic stem cell transplantation (HSCT) have improved patient outcomes but …