Target nature :
First identified as inhibiting MNBL1 and RNA (Muscleblind 1)
CAS n° :
20830-81-3
Mecanism of action :
Inhibits the interaction between MNBL1 and RNA
Physico-chemical properties :
Molecular Weight 527.5 g/mol\r\nXLogP3 1.8\r\nExact Mass 527.179146 g/mol\r\nSolubility: 39.2 mg/L\r\nSoluble in methyl alcohol, insoluble in chloroform and benzene. In water, 3.0X10+4 mg/L at 25 °C\r\npKa = 7.85
Probe availability :
Available to Prestwick Chemical company : http://www.prestwickchemical.com/index.html
Princeps reference :
Mouli Chakraborty, Chantal Sellier, Michel Ney, Pascal Villa, Nicolas Charlet-Berguerand, Ruben Artero, Beatriz Llamusi (2018) Daunorubicin reduces MBNL1 sequestration caused by CUG-repeat expansion and rescues cardiac dysfunctions in a Drosophila model of myotonic dystrophy. Disease Models & Mechanisms 2018 11: dmm032557 doi: 10.1242/dmm.032557 Published 23 April 2018
More bibliography :
Other available information on the probe :
Store at RT
Other information on the target :
The muscleblind-like (MBNL) family of proteins, comprising MBNL1, MBNL2 and MBNL3, normally bind to YGC (Y stands for pyrimidine) RNA motifs and are diverted away from their normal RNA targets by the expanded CUG RNA repeats in DMPK transcripts.\r\nReduced MBNL function appears to be a key pathogenic event underlying the skeletal muscle alterations observed in DM1. Indeed, overexpression of MBNL1 rescues DM1-like skeletal muscle alterations in a mouse model of DM1. Furthermore, compounds or strategies aimed at reducing MBNL1 binding to expanded CUG repeats alleviate skeletal muscle dysfunctions in cell and animal models of DM1.
Selectivity profile :
Not available
In vivo data :
It rescues cardiac dysfunctions in a Drosophila model of myotonic dystrophy.
In vitro data :
see Chakraborty et al (2018)
Toxicity :
Its an anticancer agent
Chaine SMILES :
COc4cccc5c(=O)c3c(O)c2C[C@@](O)(C(C)=O)C[C@H](O[C@H]1C[C@H](N)[C@H](O)[C@H](C)O1)c2c(O)c3c(=O)c45
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