ArticleJournal of neurochemistry2021
A new brain-penetrant glucosylceramide synthase inhibitor as potential Therapeutics for Gaucher disease.
Article in Journal of neurochemistry, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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11 citing papers in PubMed, 19 citations in OpenAlex.
- Assessing Surface Binding Events on Golgi Microsomes Isolated from Cancer Cells by Capillary Electrophoresis.Analytical chemistry · 2026Article
- Listening to Cellular Whispers through Glycosphingolipids: A Comprehensive Review of Recent Advancements in Glycosphingolipid Analysis and Annotation.Analytical chemistry · 2026Review
- Borylated 5-Membered Ring Iminosugars: Synthesis and Biological Evaluation for Glycosidase Inhibition and Anticancer Properties for Application in Boron Neutron Capture Therapy (BNCT)-Part 2.Pharmaceuticals (Basel, Switzerland) · 2025Article
- The SGLT2 Inhibitor Dapagliflozin Disrupts the Cell Cycle at High Concentrations Without Altering Glycosphingolipid (De Novo)Biosynthesis.International journal of molecular sciences · 2025Article
- A PIKfyve modulator combined with an integrated stress response inhibitor to treat lysosomal storage diseases.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Mysterious sphingolipids: metabolic interrelationships at the center of pathophysiology.Frontiers in physiology · 2023Review
- Juvenile mucopolysaccharidosis plus disease caused by a missense mutation in VPS33A.Human mutation · 2022Article
- Targeting the Sphingolipid Rheostat in Gliomas.International journal of molecular sciences · 2022Review
- Newborn screening for Gaucher disease in Japan.Molecular genetics and metabolism reports · 2022Article
- Drug Development in the Field of Sphinogolipid Metabolism.Advances in experimental medicine and biology · 2022Article
- A new brain-penetrant glucosylceramide synthase inhibitor as potential Therapeutics for Gaucher disease.Journal of neurochemistry · 2021Article
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gaucher disease (GD), the most common lysosomal storage disorders, is caused by GBA gene mutations resulting in glycosphingolipids accumulations in various tissues, such as the brain. While suppressing glycosphingolipid accumulation is the central strategy for treating peripheral symptoms of GD, there is no effective treatment for the central nervous system symptoms. As glycosphingolipid biosynthesis starts from ceramide glycosylation by glucosylceramide synthase (GCS), inhibiting GCS in the brain is a promising strategy for neurological GD. Herein, we discovered T-036, a potent and brain-penetrant GCS inhibitor with a unique chemical structure and binding property. T-036 does not harbor an aliphatic amine moiety and has a noncompetitive inhibition mode to the substrates, unlike other known inhibitors. T-036 exhibited sufficient exposure and a significant reduction of glucosylsphingolipids in the plasma and brain of the GD mouse model. Therefore, T-036 could be a promising lead molecule for treating central nervous system symptoms of GD.
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