Maria Prociuk
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View article: AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease) Open
Globoid cell leukodystrophy (GLD), or Krabbe disease, is an inherited, neurologic disorder that results from deficiency of a lysosomal enzyme, galactosylceramidase. Most commonly, deficits of galactosylceramidase result in widespread centr…
View article: Clinical, electrophysiological, and biochemical markers of peripheral and central nervous system disease in canine globoid cell leukodystrophy (<scp>K</scp>rabbe's disease)
Clinical, electrophysiological, and biochemical markers of peripheral and central nervous system disease in canine globoid cell leukodystrophy (<span>K</span>rabbe's disease) Open
Globoid cell leukodystrophy (GLD), or Krabbe's disease, is a debilitating and always fatal pediatric neurodegenerative disease caused by a mutation in the gene encoding the hydrolytic enzyme galactosylceramidase (GALC). In the absence of G…
611. Promoter Evaluation of AAV Gene Therapy in the Central Nervous System for Feline Niemann-Pick Type C Disease Open
Niemann-Pick type C1 (NPC1) disease is characterized by progressive cerebellar ataxia, dementia, and death in adolescence. It is caused by mutations that result in deficient function of lysosomal, membrane-bound NPC1, resulting in the intr…