Figure 1.

Recoding the GAA Sequence Increases GAA Enzyme Activity per Integration, Resulting in Increased GAA Activity in Leukocytes

(A) Gaa^−/− mice transplanted with 5 × 10⁵ Lin⁻ cells transduced with either the lentiviral vector expressing the native GAA cDNA (GAA), a codon-optimized GAA cDNA (GAAco), or GFP (control vector; KO), the transgenes driven by the SF promoter and spleen cells collected 10–12 months after transplantation from n = 3 GAA, n = 5 GAAco, and n = 3 KO mice. GAA activity per vector copy number ± SD. (B) GAA activity in leukocytes of individual mice 2 months after transplantation (GAA, n = 10; GAAco, n = 10). Significance was calculated by a Mann-Whitney U test (∗p < 0.05).