FIG. 4.

Circulating biomarker response to tissue-specific therapy. C3 (A) and MeCit (B) were assayed by tandem mass spectrometry 1 and 45 weeks after injection of 5×10¹¹ vg of AAV1-MCK-PCCA, AAV8-TTR-PCCA, AAV8-CMV-PCCA, or AAV1-MCK-PCCA/AAV8-TTR-PCCA combination treatment (n=10 each). Asterisks indicate statistical significance relative to untreated Pcca^−/−(A138T) mice at the 45-week time point. *p<0.05 and ***p<0.001. Error bars depict SEM. Color images available online at www.liebertpub.com/hum