Extended Data Fig. 2: Quantitative PCR screening of candidate PPMOs for targeted reduction of progerin in proband fibroblasts.
From: A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome
Quantitation of LMNA (A), Progerin (P), and LMNC (C) expression by ddPCR analysis of Classic HGPS (LMNA c.1824C > T) fibroblasts treated with 6uM candidate PPMOs. a, Transcript levels relative to TFRC. b, Each isoforms’ fraction relative to all LMNA transcripts. c, Each isoforms’ transcript level relative to LMNC transcripts. The greatest reduction of progerin transcripts 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 triplicate reactions of biological triplicates. * p < 0.05, ** p < 0.01, *** p < 0.001 versus transcripts or protein from cells receiving saline (NT).
