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Article

Novel compounds that reverse the disease phenotype in Type 2 Gaucher disease patient-derived cells.

Bioorganic & medicinal chemistry letters - 15 Jan 2020

Childers Wayne, Fan Rong, Martinez Rogelio, Colussi Dennis J, Melenski Edward, Liu Yuxiao, Gordon John, Abou-Gharbia Magid, Jacobson Marlene A

Abstract excerpt

Gaucher disease (GD) results from inherited mutations in the lysosomal enzyme β-glucocerobrosidase (GCase). Currently available treatment options for Type 1 GD are not efficacious for treating neuronopathic Type 2 and 3 GD due to their inability to cross the blood-brain barrier. In an effort to identify small molecules which could be optimized for CNS penetration we identified tamoxifen from a high throughput...

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Identifiers

PMID: 31757667
DOI: 10.1016/j.bmcl.2019.126806
PMCID: PMC7569734

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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