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10.1038/nrm2934 49 SieberJ.WeinsA.KampeK.GruberS.LindenmeyerM

Neonatal Screen 3, 10

To further confirm these findings, we performed Western blot and qRT-PCR analyses, which showed that both protein and mRNA levels of CPT1A were downregulated in STC-induced SAP models, both in vitro and in vivo (Figures 1DI)

We also discuss how the outcomes associated with different timing of therapeutic intervention are contributing to our understanding of the biology and pathogenesis of SMA