
Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis
Keywords: BMEF, bone marrow extracellular fluid; ERT, enzyme replacement therapy; FACE, fluorophore-assisted carbohydrate electrophoresis; GS, galactosialidosis; IHC, immunohistochemistry; KO, knockout; KO/TG, knockout–transgenic; LSD, lysosomal storage disease; PC