Matches in SemOpenAlex for { <https://semopenalex.org/work/W3195176469> ?p ?o ?g. }
- W3195176469 endingPage "553" @default.
- W3195176469 startingPage "543" @default.
- W3195176469 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." @default.
- W3195176469 created "2021-08-30" @default.
- W3195176469 creator A5001043320 @default.
- W3195176469 creator A5017848730 @default.
- W3195176469 creator A5021495006 @default.
- W3195176469 creator A5052777624 @default.
- W3195176469 creator A5060648746 @default.
- W3195176469 creator A5069235020 @default.
- W3195176469 creator A5074210227 @default.
- W3195176469 creator A5089691839 @default.
- W3195176469 date "2021-08-31" @default.
- W3195176469 modified "2023-10-17" @default.
- W3195176469 title "A new brain‐penetrant glucosylceramide synthase inhibitor as potential Therapeutics for Gaucher disease" @default.
- W3195176469 cites W1540698875 @default.
- W3195176469 cites W1601406615 @default.
- W3195176469 cites W1831130622 @default.
- W3195176469 cites W1969252444 @default.
- W3195176469 cites W1974081722 @default.
- W3195176469 cites W1986341243 @default.
- W3195176469 cites W1988843643 @default.
- W3195176469 cites W1990421239 @default.
- W3195176469 cites W1993796718 @default.
- W3195176469 cites W2017903224 @default.
- W3195176469 cites W2024179341 @default.
- W3195176469 cites W2027459860 @default.
- W3195176469 cites W2028245787 @default.
- W3195176469 cites W2041620451 @default.
- W3195176469 cites W2055445325 @default.
- W3195176469 cites W2082577333 @default.
- W3195176469 cites W2086579854 @default.
- W3195176469 cites W2110335166 @default.
- W3195176469 cites W2111930154 @default.
- W3195176469 cites W2119465020 @default.
- W3195176469 cites W2187061108 @default.
- W3195176469 cites W2229066693 @default.
- W3195176469 cites W2300525878 @default.
- W3195176469 cites W2311314554 @default.
- W3195176469 cites W2395282085 @default.
- W3195176469 cites W2466865346 @default.
- W3195176469 cites W2519587718 @default.
- W3195176469 cites W2589161214 @default.
- W3195176469 cites W2733386350 @default.
- W3195176469 cites W2739756331 @default.
- W3195176469 cites W2761470580 @default.
- W3195176469 cites W2905435570 @default.
- W3195176469 cites W2910155310 @default.
- W3195176469 cites W2913644249 @default.
- W3195176469 cites W2948188928 @default.
- W3195176469 cites W2980754698 @default.
- W3195176469 cites W2984173135 @default.
- W3195176469 cites W3073842499 @default.
- W3195176469 cites W3088857162 @default.
- W3195176469 cites W3195176469 @default.
- W3195176469 cites W2126914441 @default.
- W3195176469 doi "https://doi.org/10.1111/jnc.15492" @default.
- W3195176469 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/34398463" @default.
- W3195176469 hasPublicationYear "2021" @default.
- W3195176469 type Work @default.
- W3195176469 sameAs 3195176469 @default.
- W3195176469 citedByCount "12" @default.
- W3195176469 countsByYear W31951764692021 @default.
- W3195176469 countsByYear W31951764692022 @default.
- W3195176469 countsByYear W31951764692023 @default.
- W3195176469 crossrefType "journal-article" @default.
- W3195176469 hasAuthorship W3195176469A5001043320 @default.
- W3195176469 hasAuthorship W3195176469A5017848730 @default.
- W3195176469 hasAuthorship W3195176469A5021495006 @default.
- W3195176469 hasAuthorship W3195176469A5052777624 @default.
- W3195176469 hasAuthorship W3195176469A5060648746 @default.
- W3195176469 hasAuthorship W3195176469A5069235020 @default.
- W3195176469 hasAuthorship W3195176469A5074210227 @default.
- W3195176469 hasAuthorship W3195176469A5089691839 @default.
- W3195176469 hasBestOaLocation W31951764691 @default.
- W3195176469 hasConcept C126322002 @default.
- W3195176469 hasConcept C169760540 @default.
- W3195176469 hasConcept C178790620 @default.
- W3195176469 hasConcept C185592680 @default.
- W3195176469 hasConcept C190283241 @default.
- W3195176469 hasConcept C21850170 @default.
- W3195176469 hasConcept C2776568683 @default.
- W3195176469 hasConcept C2776931407 @default.
- W3195176469 hasConcept C2777851122 @default.
- W3195176469 hasConcept C2779134260 @default.
- W3195176469 hasConcept C2779335537 @default.
- W3195176469 hasConcept C2779969927 @default.
- W3195176469 hasConcept C529278444 @default.
- W3195176469 hasConcept C55493867 @default.
- W3195176469 hasConcept C71240020 @default.
- W3195176469 hasConcept C71924100 @default.
- W3195176469 hasConcept C86803240 @default.
- W3195176469 hasConcept C98274493 @default.
- W3195176469 hasConceptScore W3195176469C126322002 @default.
- W3195176469 hasConceptScore W3195176469C169760540 @default.
- W3195176469 hasConceptScore W3195176469C178790620 @default.
- W3195176469 hasConceptScore W3195176469C185592680 @default.
- W3195176469 hasConceptScore W3195176469C190283241 @default.
- W3195176469 hasConceptScore W3195176469C21850170 @default.
- W3195176469 hasConceptScore W3195176469C2776568683 @default.