Extended Data Fig. 4: Quantitative PCR screening of SRP-2001 for targeted reduction of progerin in Classic and Non-classic HGPS cell lines.
From: A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome
a, Transcript copy number in normal control (NL Control), Classic HGPS (c.1824C>T), and Non-classic HGPS (c.1822G>A, c.1968+1G>A, c.1968+2T>C, c.1968+5G>C) fibroblasts following 2 weeks of treatment with SRP-2001. b, Individual LMNA transcripts expressed as a fraction of all total LMNA-derived transcripts in each cell line. c, Full-length LMNA and progerin transcript levels relative to LMNC transcripts. A, LMNA; P, progerin; C, LMNC; Values and error bars represent mean ± SD determined from triplicate reactions of biological triplicates. * p < 0.05; ** p < 0.01; *** p < 0.001 vs same transcript in cells receiving saline only (NT).
