Challenges to congenital genetic disorders with "RNA-targeting" chemical compounds.
Challenges to congenital genetic disorders with "RNA-targeting" chemical compounds.
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DOI：
10.1016/j.pharmthera.2012.02.001
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发表时间：
2012-06
影响因子：
13.5
通讯作者：
Yasushi Ogawa;M. Hagiwara
中科院分区：
文献类型：
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作者：
Yasushi Ogawa;M. Hagiwara
唐氏综合症（DS）和杜氏肌营养不良症（DMD）等先天性疾病患者的染色体和/或基因存在异常。因此，人们认为药物治疗对这些患者的作用无非是在症状出现时暂时缓解症状。虽然我们不能用化学药物使他们的染色体和基因正常化，但我们也许能够用小化学物质操纵从患者 DNA 转录的 mRNA 的数量和模式。基于这个简单的想法，我们寻找可适用于先天性疾病的化合物，并发现蛋白激酶抑制剂如INDY、TG003和SRPIN340分别有望作为DS、DMD和DDS的临床药物。
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.