On the contrary, cells transduced with the dual cassette (shRNA3X and optPABPN1) were healthy and proliferating 10 days after transduction (Fig. 6b), showing that replacement of endogenous PABPN1 levels with optPABPN1 expression was sufficient to rescue cell survival of the human OPMD myoblasts. The gene discussed is PABPN1; the disease is oculopharyngeal muscular dystrophy.