... UniProtP34059), leading to lysosomal accumulation of glyco-saminoglycans, keratan sulfate and chondroitin 6-sul-fate, mainly in bone and cornea [1]. Clinicalmanifestations vary from severe to attenuated ... [27], carrying human GALNS cDNA with a CMVpromoter flanked by the inverted terminal repeats of AAV2. The pAAV–AAT–GALNS plasmid was constructedby replacement of the CMV promoter in pAAV–CMV–GALNS ... cotransduction of SUMF1.AbbreviationsAAT, a 1-antitrypsin promoter; AAV, adeno-associated virus; CMV, cytomegalovirus; EF1, elongation factor 1a; GALNS, N-acetylgalatosamine-6-sulfate sulfatase; GAPDH,...