Skip to main content
. 2015 Feb 10;23(4):638–647. doi: 10.1038/mt.2015.9

Figure 3.

Figure 3

Restoration of SGSH activity corrects behavior abnormalities and extends lifespan. MPS IIIA mice (4–6 weeks old) were treated with an intravenous injection of scAAVrh74-U1a-hSGSH vector (5 × 1012 vg/kg). (a,b) Mice were tested for behavior performance at age 7–7.7 months, in a hidden task in water maze: +/+: wt (n = 41); −/−: nontreated MPS IIIA mice (n = 33); AAV: scAAVrh74-treated MPS IIIA mice (n = 13). (c) Longevity: wt: n = 42; IIIA: n = 31; AAVrh74: n = 7. #P > 0.05 versus wt; *P ≤ 0.05 versus wt; ^P ≤ 0.05 versus nontreated IIIA. AAV, adeno-associated virus; MPS, mucopolysaccharidosis; SGSH, N-sulfoglucosamine sulfohydrolase; vg, vector genome; wt, wild-type.