Iduronidase

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  Subject Areas on Research

  • AAV Gene Therapy for MPS1-associated Corneal Blindness. 
  • Candidate gene association study for diabetic retinopathy in persons with type 2 diabetes: the Candidate gene Association Resource (CARe). 
  • Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. 
  • Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I. 
  • Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I. 
  • Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I. 
  • Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study. 
  • Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy). 
  • Rapid assays for Gaucher and Hurler diseases in dried blood spots using digital microfluidics. 
  • The North Carolina Experience with Mucopolysaccharidosis Type I Newborn Screening.