P. J. Orchard
Comparative effectiveness of intracerebroventricular, intrathecal, and intranasal routes of AAV9 vector administration for genetic therapy of neurologic disease in murine mucopolysaccharidosis type I
Frontiers in Molecular Neuroscience.
Intranasal adeno-associated virus mediated gene delivery and expression of human iduronidase in the central nervous system: a noninvasive and effective approach for prevention of neurologic disease in mucopolysaccharidosis Type I
Human Gene Therapy.
Long-term cognitive and functional outcomes in children with mucopolysaccharidosis (MPS)-IH (Hurler syndrome) treated with hematopoietic cell transplantation
Biomarkers of bone remodeling in children with mucopolysaccharidosis types I, II, and VI
Journal of Pediatric Rehabilitation Medicine.
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