Ondono, Raul published the artcileDesign of novel small molecule base-pair recognizers of toxic CUG RNA transcripts characteristics of DM1, Application of Dimethyl itaconate, the publication is Computational and Structural Biotechnology Journal (2021), 51-61, database is CAplus and MEDLINE.
Myotonic Dystrophy type 1 (DM1) is an incurable neuromuscular disorder caused by toxic DMPK transcripts that carry CUG repeat expansions in the 3′ untranslated region (3′UTR). The intrinsic complexity and lack of crystallog. data makes noncoding RNA regions challenging targets to study in the field of drug discovery. In DM1, toxic transcripts tend to stall in the nuclei forming complex inclusion bodies called foci and sequester many essential alternative splicing factors such as Muscleblind-like 1 (MBNL1). Most DM1 phenotypic features stem from the reduced availability of free MBNL1 and therefore many therapeutic efforts are focused on recovering its normal activity. For that purpose, herein we present pyrido[2,3-d]pyrimidin-7-(8H)-ones, a privileged scaffold showing remarkable biol. activity against many targets involved in human disorders including cancer and viral diseases. Their combination with a flexible linker meets the requirements to stabilize DM1 toxic transcripts, and therefore, enabling the release of MBNL1. Therefore, a set of novel pyrido[2,3-d]pyrimidin-7-(8H)-ones derivatives (1a-e) were obtained using click chem. 1a exerted over 20% MBNL1 recovery on DM1 toxic RNA activity in primary cell biol. studies using patient-derived myoblasts. 1a promising anti DM1 activity may lead to subsequent generations of ligands, highlighting a new affordable treatment against DM1.
Computational and Structural Biotechnology Journal published new progress about 617-52-7. 617-52-7 belongs to esters-buliding-blocks, auxiliary class Alkenyl,Aliphatic hydrocarbon chain,Ester, name is Dimethyl itaconate, and the molecular formula is C7H10O4, Application of Dimethyl itaconate.
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