Title : Development of an orally available inhibitor of CLK1 for skipping a mutated dystrophin exon in Duchenne muscular dystrophy.

Pub. Date : 2017 May 30

PMID : 28555643






1 Functional Relationships(s)
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1 Here, we show an orally available inhibitor of CLK1, named TG693, which promoted the skipping of the endogenous mutated exon 31 in DMD patient-derived cells and increased the production of the functional exon 31-skipped dystrophin protein. 5-Pyridin-4-yl-1H-indazole CDC like kinase 1 Homo sapiens