Extended Data Fig. 3 |. Western immunoblot screening of candidate PPMOs for targeted reduction of progerin in proband fibroblasts.

Quantitation of LMNA (A), Progerin (P), and LMNC (C) protein from immunoblots of lysates from Classic HGPS (LMNA c.1824C > T) fibroblasts treated with 6uM candidate PPMOs. Data was derived from the analysis shown in Fig. 1d and is expressed as isoform levels relative to ACTB (a), the isoforms’ fraction relative to all LMNA gene products (b), and the isoforms’ level relative to LMNC protein (c). The greatest reduction of progerin protein in culture was achieved with the centrally located morpholino (SRP-2001). A, LMNA; P, progerin; C, LMNC; WT, oligo complementary to normal LMNA sequence; CTRL, scrambled control; Values and error bars represent mean ± SD determined from biological triplicates shown in Fig. 1d. * p < 0.05, ** p < 0.01, *** p < 0.001 versus transcripts or protein from cells receiving saline NT).