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. 2018 Mar 16;9:234–246. doi: 10.1016/j.omtm.2018.03.004

Figure 6.

Figure 6

IV Delivery of rAAV9.HR-ASPA at P1 to CD Mice Corrected Brain Pathology and Extended Survival as Efficiently as rAAV9wt-ASPA

(A) Coronal series of T2-weighted MRI brain images obtained from an untreated wild-type mouse (WT, P42) and Canavan disease (CD) mice treated with 0.9% NaCl (NaCl, P27), rAAV9wt-ASPA (rAAV9wt, P42), or rAAV9.HR-ASPA (rAAV9.HR, P42). Note that treatment with either rAAV9wt or rAAV9.HR in CD mice completely normalized the hyperintensity seen in the NaCl-treated CD mouse. (B) Bar graph shows NAA levels normalized to creatine levels (tNAA/tCr; determined by MRS) in the brains of CD mice treated as described in (A) (n = 3). **p < 0.01 and ns, not significant by Tukey’s multiple comparisons test following one-way ANOVA. Dashed line indicates WT level. Data are presented as mean and SD. (C) Kaplan-Meier survival curves show the percentage of survived mice over time course, including CD mice that were left untreated (red, n = 12), treated with rAAV9wt-ASPA (gray, n = 12), or treated with rAAV9.HR-ASPA (purple, n = 13).