Figure 6: PABPN1 inhibition in human OPMD myoblasts induces cell death that can be prevented by optPABPN1 co-expression.
From: PABPN1 gene therapy for oculopharyngeal muscular dystrophy
(a,b) Primary OPMD myoblast cells were transduced with shRNA3X tricistronic cassette, shRNA-HBVpol, shRNA3X-optPABPN1 or shRNA HBVpol-optPABPN1 LV vectors. GFP observed 4 and 10 days post transduction demonstrates that no GFP+ cells survived on the condition transduced with shRNA3X. However, co-transduction with shRNA-HBVpol-optPABPN1 prevented cell death. Bar, 100 μm.
