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. Author manuscript; available in PMC: 2013 Feb 1.
Published in final edited form as: Mol Genet Metab. 2011 Nov 7;105(2):203–211. doi: 10.1016/j.ymgme.2011.10.020

Figure 4. Dose response study on the efficacy of AAV2/8sc.TBG.hOTCco vector.

Figure 4

Adult male spfash mice were injected i.v. with AAV2/8sc.TBG.hOTCco vector at the dose of 1×1011, 3×1010, 1×1010, 3×109 and 1×109 GC. (a) Robust and sustained normalization of urinary orotic acid levels following vector treatment. Shaded area indicates orotic acid levels in normal mice. (b) Outcome of NH4Cl challenge performed at 4, 6, 13, 23, or 40 weeks post AAV vector treatment.