ArticlePloS one2015
Tool compounds robustly increase turnover of an artificial substrate by glucocerebrosidase in human brain lysates.
Article in PloS one, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
5 citing papers in PubMed, 13 citations in OpenAlex.
- Article
- High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Article
- In situ visualization of glucocerebrosidase in human skin tissue: zymography versus activity-based probe labeling.Journal of lipid research · 2017Article
- Design and Synthesis of Potent Quinazolines as Selective β-Glucocerebrosidase Modulators.Journal of medicinal chemistry · 2016Article
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Authors and funding
8 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Mutations in glucocerebrosidase (GBA1) cause Gaucher disease and also represent a common risk factor for Parkinson's disease and Dementia with Lewy bodies. Recently, new tool molecules were described which can increase turnover of an artificial substrate 4MUG when incubated with mutant N370S GBA1 from human spleen. Here we show that these compounds exert a similar effect on the wild-type enzyme in a cell-free system. In addition, these tool compounds robustly increase turnover of 4MUG by GBA1 derived from human cortex, despite substantially lower glycosylation of GBA1 in human brain, suggesting that the degree of glycosylation is not important for compound binding. Surprisingly, these tool compounds failed to robustly alter GBA1 turnover of 4MUG in the mouse brain homogenate. Our data raise the possibility that in vivo models with humanized glucocerebrosidase may be needed for efficacy assessments of such small molecules.
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Registered trials
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