Figure 1.

AAV9 galactosylceramidase vector engineering. (A) Schematic outline of the vector. CAG, cytomegalovirus early enhancer/chicken β-actin promoter; hIDS-SP, the coding sequence of the signal peptide of the human iduronate-2-sulfatase gene; mGALC, the codon-optimized mouse galactosylceramidase gene coding sequence; hApoB-LDLR-BD, the coding sequence of the low-density lipoprotein receptor binding domain of the human apolipoprotein B gene; pA, polyadenylation signal. (B) Representative galactosylceramidase enzymatic staining photomicrographs from a 3-month-old untreated mouse (top panels) and a 3-month-old mouse that had received intravenous injection of the AAV9 galactosylceramidase vector at 2 days of age (bottom panels). Systemic injection resulted in widespread galactosylceramidase expression in multiple organs. AAV9, adeno-associated virus serotype 9.