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This precision medicine disarms the disease at the genetic level by hybridizing with SOD1 mRNA, orchestrating its degradation and consequently snuffing out the production of the detrimental SOD1 protein (8, 43, 44)

Neuron 74 , 691705 (2012)

Cancer 12 , 63 (2013)

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Moreover, consistent with the inhibition of constitutive STAT3 functions, treatment with the R001 attenuated the expression of STAT3 downstream target genes, including c-Myc, Mcl-1, Bcl-2, Bcl-xL, and vascular endothelial growth factor (VEGF) in MDA-MB-468 and MDA-MB-231 cells (Fig