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. Author manuscript; available in PMC: 2017 May 1.
Published in final edited form as: Exp Neurol. 2016 Feb 15;279:13–26. doi: 10.1016/j.expneurol.2016.02.009

Figure 7. The effects of 4PBA and VX563 on SMN expression and function in the spinal cords of SMNΔ7 SMA mice.

Figure 7

SMNΔ7 SMA mice (n = 3/group) were treated with either 4PBA, VX563 or their appropriate vehicles for 5 days beginning at PND04. Age-matched carrier mice were also included as controls. Neither 4PBA nor VX563 significantly affected the levels of FLSMN (A) mRNAs in treated spinal cord samples as determined by qRT-PCR. Immunoblot analysis shows that the levels of total SMN protein in the spinal cord were not altered by treatment with either 4PBA (B) or VX563 (C). Quantification of human SMN protein levels by ELISA also shows no significant changes in response to 4PBA or VX563 treatment (D). In vitro U1 snRNP assembly was not altered in spinal cord samples from SMNΔ7 SMA mice treated with either 4PBA or VX563 relative to vehicle-treated SMNΔ7 SMA mice (E). The asterisk (*) denotes a statistically significant (p ≤ 0.05) difference when compared to vehicle-treated SMNΔ7 SMA mice for each drug.