BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

223 related articles for article (PubMed ID: 21738789)

  • 1. Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.
    Ashe KM; Bangari D; Li L; Cabrera-Salazar MA; Bercury SD; Nietupski JB; Cooper CG; Aerts JM; Lee ER; Copeland DP; Cheng SH; Scheule RK; Marshall J
    PLoS One; 2011; 6(6):e21758. PubMed ID: 21738789
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.
    Nietupski JB; Pacheco JJ; Chuang WL; Maratea K; Li L; Foley J; Ashe KM; Cooper CG; Aerts JM; Copeland DP; Scheule RK; Cheng SH; Marshall J
    Mol Genet Metab; 2012 Apr; 105(4):621-8. PubMed ID: 22366055
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity.
    Marshall J; Nietupski JB; Park H; Cao J; Bangari DS; Silvescu C; Wilper T; Randall K; Tietz D; Wang B; Ying X; Leonard JP; Cheng SH
    Mol Ther; 2019 Aug; 27(8):1495-1506. PubMed ID: 31208914
    [TBL] [Abstract][Full Text] [Related]  

  • 4. N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice.
    Baek RC; Kasperzyk JL; Platt FM; Seyfried TN
    Neurochem Int; 2008 May; 52(6):1125-33. PubMed ID: 18207611
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin.
    Jeyakumar M; Butters TD; Cortina-Borja M; Hunnam V; Proia RL; Perry VH; Dwek RA; Platt FM
    Proc Natl Acad Sci U S A; 1999 May; 96(11):6388-93. PubMed ID: 10339597
    [TBL] [Abstract][Full Text] [Related]  

  • 6. A novel glucosylceramide synthase inhibitor attenuates alpha synuclein pathology and lysosomal dysfunction in preclinical models of synucleinopathy.
    Cosden M; Jinn S; Yao L; Gretzula CA; Kandebo M; Toolan D; Hatcher NG; Ma L; Lemaire W; Adam GC; Burlein C; Minnick C; Flick R; Watt ML; Mulhearn J; Fraley M; Drolet RE; Marcus JN; Smith SM
    Neurobiol Dis; 2021 Nov; 159():105507. PubMed ID: 34509608
    [TBL] [Abstract][Full Text] [Related]  

  • 7. A new brain-penetrant glucosylceramide synthase inhibitor as potential Therapeutics for Gaucher disease.
    Fujii T; Tanaka Y; Oki H; Sato S; Shibata S; Maru T; Tanaka Y; Tanaka M; Onishi T
    J Neurochem; 2021 Nov; 159(3):543-553. PubMed ID: 34398463
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis.
    Elliot-Smith E; Speak AO; Lloyd-Evans E; Smith DA; van der Spoel AC; Jeyakumar M; Butters TD; Dwek RA; d'Azzo A; Platt FM
    Mol Genet Metab; 2008 Jun; 94(2):204-11. PubMed ID: 18387328
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.
    Jeyakumar M; Norflus F; Tifft CJ; Cortina-Borja M; Butters TD; Proia RL; Perry VH; Dwek RA; Platt FM
    Blood; 2001 Jan; 97(1):327-9. PubMed ID: 11133779
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.
    Butters TD; Dwek RA; Platt FM
    Glycobiology; 2005 Oct; 15(10):43R-52R. PubMed ID: 15901676
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Restricted ketogenic diet enhances the therapeutic action of N-butyldeoxynojirimycin towards brain GM2 accumulation in adult Sandhoff disease mice.
    Denny CA; Heinecke KA; Kim YP; Baek RC; Loh KS; Butters TD; Bronson RT; Platt FM; Seyfried TN
    J Neurochem; 2010 Jun; 113(6):1525-35. PubMed ID: 20374428
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Substrate deprivation therapy in juvenile Sandhoff disease.
    Wortmann SB; Lefeber DJ; Dekomien G; Willemsen MA; Wevers RA; Morava E
    J Inherit Metab Dis; 2009 Dec; 32 Suppl 1():S307-11. PubMed ID: 19898952
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Ethylenedioxy-PIP2 oxalate reduces ganglioside storage in juvenile Sandhoff disease mice.
    Arthur JR; Wilson MW; Larsen SD; Rockwell HE; Shayman JA; Seyfried TN
    Neurochem Res; 2013 Apr; 38(4):866-75. PubMed ID: 23417430
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis.
    Jeyakumar M; Butters TD; Dwek RA; Platt FM
    Neuropathol Appl Neurobiol; 2002 Oct; 28(5):343-57. PubMed ID: 12366816
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Substrate reduction therapy of glycosphingolipid storage disorders.
    Aerts JM; Hollak CE; Boot RG; Groener JE; Maas M
    J Inherit Metab Dis; 2006; 29(2-3):449-56. PubMed ID: 16763917
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease.
    Andersson U; Smith D; Jeyakumar M; Butters TD; Borja MC; Dwek RA; Platt FM
    Neurobiol Dis; 2004 Aug; 16(3):506-15. PubMed ID: 15262262
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Glycosphingolipid degradation and animal models of GM2-gangliosidoses.
    Kolter T; Sandhoff K
    J Inherit Metab Dis; 1998 Aug; 21(5):548-63. PubMed ID: 9728335
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Glycosphingolipid storage leads to the enhanced degradation of the B cell receptor in Sandhoff disease mice.
    te Vruchte D; Jeans A; Platt FM; Sillence DJ
    J Inherit Metab Dis; 2010 Jun; 33(3):261-70. PubMed ID: 20458542
    [TBL] [Abstract][Full Text] [Related]  

  • 19. CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.
    Marshall J; Sun Y; Bangari DS; Budman E; Park H; Nietupski JB; Allaire A; Cromwell MA; Wang B; Grabowski GA; Leonard JP; Cheng SH
    Mol Ther; 2016 Jun; 24(6):1019-1029. PubMed ID: 26948439
    [TBL] [Abstract][Full Text] [Related]  

  • 20. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease.
    McEachern KA; Fung J; Komarnitsky S; Siegel CS; Chuang WL; Hutto E; Shayman JA; Grabowski GA; Aerts JM; Cheng SH; Copeland DP; Marshall J
    Mol Genet Metab; 2007 Jul; 91(3):259-67. PubMed ID: 17509920
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 12.