FIGURE 5
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Long-Term Correction of Ornithine Transcarbamylase Deficiency by WPRE-Mediated Overexpression Using a Helper-Dependent Adenovirus
Asad Mian, W. Michael McCormack Jr., Viraj Mane, Soledad Kleppe, Philip Ng, Milton Finegold, William E. O'Brien, John R. Rodgers, Arthur L. Beaudet and Brendan Lee
BACK TO ARTICLEFigure 5.
Increased steady-state levels of OTC mRNA and protein in mice treated with HDV-hOTC-WPRE. (A) Northern blot. The relative intensities of hOTC mRNA and mouse Gapdh were quantified at week 5 only. At steady state, the amount of hOTC mRNA was significantly elevated in the livers of spf-ash mice treated with the WPRE-containing vector compared with the other group (* P < 0.0005 for week 5). 1, PBS-treated mice; 2 and hO, HDV-PEPCK-hOTC–treated mice; 3 and hO-W, HDV-PEPCK-hOTC-WPRE–treated mice. (B) Western blot. hOTC (lanes in groups 2 and 3) and mOTC (lanes in group 1) were detected by a polyclonal antibody generated to hOTC. The OTC protein was normalized to an internal control band (int. cont.). At steady state, the amount of hOTC protein was significantly elevated in the livers of spf-ash mice treated with HDV-PEPCK-hOTC-WPRE (* P < 0.02 for week 5). 1, PBS-treated mice; 2, and hO, HDV-PEPCK-hOTC–treated mice; 3 and hO-W, HDV-PEPCK-hOTC-WPRE–treated mice.
