Figure 1.

Intraparenchymal Injection of LYS-SAF302 in MPS IIIA Mice

The injection sites (white arrows) are shown from a lateral perspective and the locations of hemi-coronal brain slices 1–5 are indicated. MPS IIIA mice received stereotactic injection of LYS-SAF302 at 5 weeks of age using a Hamilton syringe (n = 10/sex/group). Vectors were administered at a dose of 8.6E+08 vg (low dose), 4.1E+10 vg (medium dose), or 9.0E+10 vg (high dose) in 8 μL delivered at 0.2 μL/min via 2 μL into each of the left and right striatum and 2 μL into each of the left and right thalamus.