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. Author manuscript; available in PMC: 2023 May 4.
Published in final edited form as: Nat Med. 2021 Mar 11;27(3):536–545. doi: 10.1038/s41591-021-01274-0

Fig. 3 |. Determination of SRP-2001 efficacy in culture.

Fig. 3 |

a–f, Fibroblasts derived from a proband with classic HGPS (a–c) and mice carrying two copies of the human mutant LMNA transgene (LMNAG/G) (d–f) were treated for two weeks with SRP-2001 and analyzed for LMNA expression by qPCR and western immunoblot. For transcript quantitation, the averages and s.d. were determined from triplicate reactions of biological triplicates. The values for protein analyses were determined from three independent cultures. The EC50 value was estimated at 3 μM based on intracellular progerin transcript (a) and protein (b,c) levels in HGPS cells and progerin transcript (d) and protein (e,f) levels in transgenic mouse cells. *P < 0.05; **P < 0.01; ***P < 0.001 versus the same transcripts or proteins in cells receiving saline only.