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

Abstract:

: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.

journal_name

Pharmacol Ther

authors

Ogawa Y,Hagiwara M

doi

10.1016/j.pharmthera.2012.02.001

subject

Has Abstract

pub_date

2012-06-01 00:00:00

pages

298-305

issue

3

eissn

0163-7258

issn

1879-016X

pii

S0163-7258(12)00031-9

journal_volume

134

pub_type

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