Extended Data Fig. 8: SRP-2001 reduces expression of transgene-derived progerin in murine liver.
From: A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome
a, Quantitative PCR analysis of transgene transcripts in liver tissue of LMNAG/G mice receiving long-term treatment with saline (vehicle) or 60 mg/kg SRP-2001. Expression of LMNA (A), progerin (P) and LMNC (C) is quantitated relative to Hprt expression, as a fraction of all transgene-derived LMNA transcripts or relative to transgene-derived LMNC transcripts. Data are presented as mean values ± SD determined from 12 independent samples per treatment group (n = 6 males, 6 females). b, Western immunoblots of A-type lamins and beta actin (ACTB) immunoprecipitated from liver homogenates. c, Quantitation of A-type lamins from immunoblots expressed relative to beta actin (ACTB), as a fraction of all A-type lamins and relative to LMNC. Data are presented as mean values ± SD determined from 12 independent samples per treatment group (n = 6 males, 6 females), shown in panel b. * p < 0.05; ** p < 0.01; *** p < 0.001 vs same transcript or protein in mice receiving saline only (vehicle).
