Figure 6. In vivo gene transfer of a HDA encoding PBGD is inhibited by monoclonal and polyclonal IgM.

Gene transfer with a HDA vector intended for the correction of acute intermittent porphyria were performed in Rag2KO mice or control WT mice. Immunoglobulins were injected 24 h and 2 h before the infusion of 5×10¹⁰ vp/mouse of HDA-PBGD. PBGD enzymatic activity in liver homogenates obtained 48 h after gene transfer (left panel) and PBGD transgene relative DNA content to GADPH by quantitative PCR (right panel). WT, Wild-type; Rag2KO, Rag^−/−IL-2Rγ^−/−; pIgM, polyclonal immunoglobulin M; mIgM, monoclonal immunoglobulin M, mIgG, monoclonal immunoglobulin G; HDA-PBGD, helper-dependent adenoviral vectors encoding human porphobilinogen deaminase.