... caused by deficiency of N-acetylgalatosamine-6-sulfate sulfatase (GALNS; EC 3.1.6.4, UniProtP34059), leading to lysosomal accumulation of glyco-saminoglycans, keratan sulfate and chondroitin ... AAV, adeno-associated virus; CMV, cytomegalovirus; EF1, elongation factor 1a; GALNS, N-acetylgalatosamine-6-sulfate sulfatase; GAPDH, glyceraldehyde-3-phosphate dehydrogenase; IRES, internal ribosomal ... (MPS) IVA is an autosomal recessive disordercaused by deficiency of the lysosomal enzyme N-acetylgalatosamine-6-sul-fate sulfatase (GALNS), which leads to the accumulation of keratan sulfateand...