AAV9/SLC6A1 gene therapy rescues abnormal EEG patterns and cognitive behavioral deficiencies in Slc6a1-/- mice.

Neither vector significantly improved the reduced BW, fear conditioning, center duration of open field, rotarod performance, or the hind limb clasping phenotype in Slc6a1 –/– KO mice ( Supplemental Figure 4 ), However, in rotarod tests, the JeT vector at a low dose (5.6 × 10 11 vg) showed a trend toward increasing rotarod performance in treated Slc6a1 –/– KO mice, but this was not observed in the high-dose JeT treatment group ( Supplemental Figure 4 , F and G).

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