Figure 5.

Gapmer AON Decreased Intracellular Collagen VI Retention and Increased Its ECM Deposition in Fibroblasts

The fibroblasts were permeabilized by 0.1% Triton in blocking buffer during immunostaining. Strong intracellular accumulation was observed in patient’s untreated fibroblasts (UCMD) compared to healthy control fibroblasts, where collagen VI was localized in the ECM. After a single treatment of AON-2 (UCMD+AON-2) or AON-3 (UCMD+AON-3) at 20 nM, the retention of intracellular collagen VI was markedly reduced, although it was notable occasionally in some cells (asterisk) and accompanied by partial deposition in the ECM (arrow).