Abstract:BackgroundFabry disease (FD) is an inherited lysosomal storage disease caused by deficiency of α‐galactosidase A (α‐Gal A) encoded by the GLA gene. The symptoms of FD occur as a result of the accumulation of globotriaosylceramide (Gb3), comprising a substrate of α‐Gal A, in the organs. Adeno‐associated virus (AAV)‐mediated gene therapy is a promising treatment for FD.Methodsα‐Gal A knockout (GLAko) mice were injected intravenously with AAV2 (1 × 1011 viral genomes [vg]) or AAV9 (1 × 1011 or 2 × 1012 vg) vector… Show more
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