Fig.4.

AON-treated patients cells are resistant to hypo-osmotic shock. (A) Membrane rupture in response to hypo-osmotic shock was assessed using calcein-AM to distinguish between intact and burst cells. Low-magnification epifluorescence snapshots of calcein-AM in mock and treated cells at the beginning (t = 0 s, top) and at the end (t = 450 sec, bottom) of the hypo-osmotic shock (Bar = 100 μm). (B) Plot of final/initial fluorescence intensity ratio for control myotubes (WT, n = 74, dark dots), for patient 2 myotubes untreated (MOCK, n = 27, white triangles), treated with AON D (n = 31, dark triangles) during the hypo-osmotic shock. The bursting fraction corresponds to the cell population whose fluorescence ratio is below the selected bursting threshold. The bursting threshold has been set to 0.5 of normalized fluorescence intensity (horizontal red line).