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Pharmacol Ther. 2012 Jun;134(3):298-305. doi: 10.1016/j.pharmthera.2012.02.001. Epub 2012 Feb 9.

Challenges to congenital genetic disorders with "RNA-targeting" chemical compounds.

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Department of Dermatology, Nagoya University Graduate School of Medicine, Tsurumai 65, Nagoya 466-8550, Japan.


Patients of congenital diseases such as Down syndrome (DS) and Duchenne muscular dystrophy (DMD) have abnormalities in their chromosomes and/or genes. Therefore, it has been considered that drug treatments can serve to do little for these patients more than to patch over each symptom temporarily when it arises. Although we cannot normalize their chromosomes and genes with chemical drugs, we may be able to manipulate the amounts and patterns of mRNAs transcribed from patients' DNAs with small chemicals. Based on this simple idea, we have looked for chemical compounds which can be applicable for congenital diseases and found that protein kinase inhibitors such as INDY, TG003, and SRPIN340 are promising as clinical drugs for DS, DMD, and DDS, respectively.

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