GTMB 7 - Gene Therapy & Molecular Biology

Gene Therapy and Molecular Biology Vol 7, page 201Figure 8: Durability of expression and tolerance of exogenous and expressed hFIX. Prenatal and adult mice were injected intravenouslywith adenoviral vectors expressing the hFIX gene (AdhFIX) and repeatedly rechallenged, as adults, with intraperitoneal hFIX proteinthen intravenous AdhFIX while hFIX concentrations were measured. The y axis shows blood hFIX concentrations (µg/ml) after in uteroor adult injection of AdhFIX (Phase I), repeated injection of hFIX protein to the adult mice (Phase II) and repeated injection of AdhFIXto the adult mice (Phase III). The x-axis shows the experimental time course in days. Arrows indicate injection points. Groups I and IIare mice initially injected in utero with AdhFIX at days 15 and 17 of gestation, respectively. Group III contains mice initially injectedintravenously with AdhFIX as adults. Group IV did not receive prior injection of AdhFIX. Group V received neither prior injections ofAdhFIX or hFIX protein. A line representing a therapeutic threshold of 40 ng/ml hFIX is included. Points are mean±S.D. (Waddingtonet al 2002). Reprinted with permission from the American Society of Hematology.201