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Browsing Auburn Theses and Dissertations by Author "Maguire, Anne"
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Myelin Deficits and Intravenous Gene Therapy in Feline Sandhoff Disease
Maguire, Anne (2021-07-27)
Sandhoff Disease (SD) is a neurodegenerative lysosomal storage disease (LSD) that results in the death of children before 4 years of age. Because there are no FDA-approved therapies available, current treatment strategies ...