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» AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease
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Endocrine/Metabolic
AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease
May 25, 2023
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Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the
GLA
gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.
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