Signal Peptide Engineering and Codon Optimization to Enhance α-Gal A Activity for rAAV Gene Therapy of Fabry Disease.

The reduction of Lyso‐Gb3 in plasma and peripheral tissues showed a trend toward being greater in the scAAV8‐sp21‐hGLAco group (Figure 4E ), which is consistent with the corresponding enzyme activity measurements (Figure 4C ), although a definitive conclusion could not be drawn due to the low absolute Lyso‐Gb3 levels for most groups.

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