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Synergistic neuroprotective effect via simian lentiviral vector‐mediated simultaneous gene transfer of human pigment epithelium‐derived factor and human fibroblast growth factor‐2 in rodent models of retinitis pigmentosa

We previously demonstrated that a new lentiviral vector derived from nonpathogenic simian immunodeficiency virus (SIVagm) was efficient and safe for long‐lasting retinal gene transfer, and that it provided the significant therapeutic effect of expressing human pigment epithelium‐derived factor (hPEDF) in Royal College of Surgeons (RCS) rats. In the present study, to obtain a more pronounced outcome, we assessed the potential synergistic effect of the simultaneous gene transfer of hPEDF and human fibroblast growth factor‐2 (hFGF‐2) by improved third‐generation SIV on RCS rats and retinal degeneration slow (rds) mice, because the former targets the primary neurons, including photoreceptor cells (PCs), whereas the latter is effective for targeting secondary neural cells, including Muller cells.