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.