Figure 5. Effect of microRNA target sites on localization and expression level following IM injection of AAV8 in mice.

Differential ffLuc expression patterns were visualized on day 7 post-IM administration of 10¹⁰ GC AAV8.CMV.ffLuc (A), AAV8.CMV.ffLuc.miR-122 (B) or AAV8.CMV.ffLuc.miR-206 (C). ffLuc expressing vectors were administered to C57BL/6 mice in two 10 µl injections into the right and left gastrocnemius muscles. (D) Liver and muscle expression of ffLuc was quantified separately on day 28 post-vector administration. Following substitution of ffLuc for 201Ig IA, expression of 201Ig IA was determined in RAG KO mice. Mice were injected with 10¹¹ GC of vectors, administered as described previously and expression of 201Ig IA in serum was measured by ELISA (E). GC of the 201Ig IA vectors present in the liver of RAG KO mice at day 90 post-vector administration was determined (F). Values are expressed as mean ± SEM (n = 3/group). *p<0.05, ***p<0.001.