Figure 4.

Recombinant human GNE mRNA expression post intravenous injection. Mice were administered a single intravenous injection of 0 μg, 10 μg, 40 μg, or 100 μg GNE-lipoplex. At the end of 2 weeks major organs (kidney, spleen, liver, and lungs) were collected. Total RNA was extracted and real-time qPCR analysis was performed. A) The percentage of mice expressing rGNE mRNA per tissue is dose dependant, with at least 75% of the animals expressing rGNE mRNA at the 100 μg dose. B) Dose dependant rGNE mRNA expression, predominantly in the lungs and liver.