RECTAS, a pre-mRNA splice modulator (Rectifier of Aberrant Splicing), was discovered in a small molecule screen for compounds that rectify aberrant IKBKAP splicing in cells from patients with familial dysautonomia. RECTAS (2 mM) increased the expression of IKAP and restored tRNA modifications.1,2 It directly interacts with CDC-like kinases enhancing SRSF6 phosphorylation.2 A combination treatment with RECTAS and phenylbutyric acid was shown to restore DJ-1 protein and mitochondrial dysfunction in Parkinsons disease models.3
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