Fig. 6: AAV-K55-GRDC24 showed anti-tumor efficacies in 2 NF1 xenograft models.

A Efficacies of AAV K55-GRDC24 in ST88-14 xenograft tumor. NSG mice were implanted with ST88-14 tumor cells in the sciatic nerve and after 2 weeks, one dose of 10¹² AAV was injected IV. Tumor growth was monitored via IVIS imaging and showed significant inhibition by AAV-K55-GRDC24 compared to the untreated control. However, after four weeks, the effects of the treatment became insignificant (n = 5 mice). Data are presented as mean values with SD and analyzed by one-tailed t-test. B In ST88-14 xenograft tumor model, two doses of AAV K55-GRDC24 enabled a more sustained response. In a procedure similar to A, a second dose of 10¹² AAV was injected IV one week after the first dose (n = 4 mice, Con: untreated control). Data are presented as mean values with SD and analyzed by one-tailed t-test. C Comparing the payload expression of one dose vs two doses AAV-K55-GFP. AAV-K55-GFP was injected IV in the ST88-14 tumor-bearing mice at a dose of 10¹² vg. Another dose was given in the double dose cohort 5 days later. All tumors were harvested 14 days after the initial dose and Western blotting indicated the enhanced expression of GFP in the tumors treated with two doses. D Efficacies of AAV K55-GRDC24 in RHT92 xenograft tumor. One dose of 10¹² AAV was injected IV one month after the tumor implantation in the sciatic nerve (n = 5 mice, Con: untreated control). Data are presented as mean values with SD and analyzed by one-tailed t-test. E In RHT92 xenograft tumor model, two doses of AAV K55-GRDC24 resulted in a substantial response. In a procedure similar to C, a second dose of 10¹² AAV was injected IV 5 days after the first dose (n = 4 mice, Con: untreated control). Data are presented as mean values with SD and analyzed by one-tailed t-test.