BzDANP, a Small-Molecule Modulator of Pre-miR-29a Maturation by Dicer

2016 ◽  
Vol 11 (10) ◽  
pp. 2790-2796 ◽  
Author(s):  
Asako Murata ◽  
Takahiro Otabe ◽  
Jinhua Zhang ◽  
Kazuhiko Nakatani
Keyword(s):  
Small Molecule ◽  
Small Molecule Modulator

scholarly journals A small-molecule modulator of cardiac myosin acts on multiple stages of the myosin chemomechanical cycle

2017 ◽  
Vol 292 (40) ◽  
pp. 16571-16577 ◽  
Author(s):  
Raja F. Kawas ◽  
Robert L. Anderson ◽  
Sadie R. Bartholomew Ingle ◽  
Yonghong Song ◽  
Arvinder S. Sran ◽  
...  
Keyword(s):  
Small Molecule ◽  
Cardiac Myosin ◽  
Small Molecule Modulator ◽  
Multiple Stages

scholarly journals A modulator of wild-type glucocerebrosidase improves pathogenic phenotypes in dopaminergic neuronal models of Parkinson’s disease

2019 ◽  
Vol 11 (514) ◽  
pp. eaau6870 ◽  
Author(s):  
Lena F. Burbulla ◽  
Sohee Jeon ◽  
Jianbin Zheng ◽  
Pingping Song ◽  
Richard B. Silverman ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Small Molecule ◽  
Dopaminergic Neurons ◽  
Wild Type ◽  
Gene Encoding ◽  
Alternative Approach ◽  
Consequent Reduction ◽  
Small Molecule Modulator ◽  
Small Molecule Modulators

Mutations in the GBA1 gene encoding the lysosomal enzyme β-glucocerebrosidase (GCase) represent the most common risk factor for Parkinson’s disease (PD). GCase has been identified as a potential therapeutic target for PD and current efforts are focused on chemical chaperones to translocate mutant GCase into lysosomes. However, for several GBA1-linked forms of PD and PD associated with mutations in LRRK2, DJ-1, and PARKIN, activating wild-type GCase represents an alternative approach. We developed a new small-molecule modulator of GCase called S-181 that increased wild-type GCase activity in iPSC-derived dopaminergic neurons from sporadic PD patients, as well as patients carrying the 84GG mutation in GBA1, or mutations in LRRK2, DJ-1, or PARKIN who had decreased GCase activity. S-181 treatment of these PD iPSC-derived dopaminergic neurons partially restored lysosomal function and lowered accumulation of oxidized dopamine, glucosylceramide and α-synuclein. Moreover, S-181 treatment of mice heterozygous for the D409V GBA1 mutation (Gba1D409V/+) resulted in activation of wild-type GCase and consequent reduction of GCase lipid substrates and α-synuclein in mouse brain tissue. Our findings point to activation of wild-type GCase by small-molecule modulators as a potential therapeutic approach for treating familial and sporadic forms of PD that exhibit decreased GCase activity.

scholarly journals Characterization of a Small‐molecule Modulator of IRE1α Activity

2015 ◽  
Vol 29 (S1) ◽  
Author(s):  
Jemilat Salami‐Oyenuga ◽  
Kanak Raina ◽  
Craig Crews
Keyword(s):  
Small Molecule ◽  
Small Molecule Modulator

Discovery of small-molecule modulator of heterotrimeric Gi-protein by integrated phenotypic profiling and chemical proteomics

2020 ◽  
Vol 84 (12) ◽  
pp. 2484-2490
Author(s):  
Tatsuro Kawamura ◽  
Yushi Futamura ◽  
Erchang Shang ◽  
Makoto Muroi ◽  
Petra Janning ◽  
...  
Keyword(s):  
Small Molecule ◽  
Chemical Proteomics ◽  
Gi Protein ◽  
Small Molecule Modulator
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