BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

252 related articles for article (PubMed ID: 19059399)

  • 1. Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease.
    Dodge JC; Clarke J; Treleaven CM; Taksir TV; Griffiths DA; Yang W; Fidler JA; Passini MA; Karey KP; Schuchman EH; Cheng SH; Shihabuddin LS
    Exp Neurol; 2009 Feb; 215(2):349-57. PubMed ID: 19059399
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse.
    Yang WW; Dodge JC; Passini MA; Taksir TV; Griffiths D; Schuchman EH; Cheng SH; Shihabuddin LS
    Exp Neurol; 2007 Oct; 207(2):258-66. PubMed ID: 17686472
    [TBL] [Abstract][Full Text] [Related]  

  • 3. AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease.
    Passini MA; Macauley SL; Huff MR; Taksir TV; Bu J; Wu IH; Piepenhagen PA; Dodge JC; Shihabuddin LS; O'Riordan CR; Schuchman EH; Stewart GR
    Mol Ther; 2005 May; 11(5):754-62. PubMed ID: 15851014
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Neuropathology of the acid sphingomyelinase knockout mouse model of Niemann-Pick A disease including structure-function studies associated with cerebellar Purkinje cell degeneration.
    Macauley SL; Sidman RL; Schuchman EH; Taksir T; Stewart GR
    Exp Neurol; 2008 Dec; 214(2):181-92. PubMed ID: 18778708
    [TBL] [Abstract][Full Text] [Related]  

  • 5. AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease.
    Barbon CM; Ziegler RJ; Li C; Armentano D; Cherry M; Desnick RJ; Schuchman EH; Cheng SH
    Mol Ther; 2005 Sep; 12(3):431-40. PubMed ID: 16099409
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Injection of mouse and human neural stem cells into neonatal Niemann-Pick A model mice.
    Sidman RL; Li J; Stewart GR; Clarke J; Yang W; Snyder EY; Shihabuddin LS
    Brain Res; 2007 Apr; 1140():195-204. PubMed ID: 17289003
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease.
    Dodge JC; Clarke J; Song A; Bu J; Yang W; Taksir TV; Griffiths D; Zhao MA; Schuchman EH; Cheng SH; O'Riordan CR; Shihabuddin LS; Passini MA; Stewart GR
    Proc Natl Acad Sci U S A; 2005 Dec; 102(49):17822-7. PubMed ID: 16301517
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease.
    Miranda SR; Erlich S; Friedrich VL; Gatt S; Schuchman EH
    Gene Ther; 2000 Oct; 7(20):1768-76. PubMed ID: 11083499
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Infusion of recombinant human acid sphingomyelinase into niemann-pick disease mice leads to visceral, but not neurological, correction of the pathophysiology.
    Miranda SR; He X; Simonaro CM; Gatt S; Dagan A; Desnick RJ; Schuchman EH
    FASEB J; 2000 Oct; 14(13):1988-95. PubMed ID: 11023983
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Identification of novel biomarkers for Niemann-Pick disease using gene expression analysis of acid sphingomyelinase knockout mice.
    Dhami R; Passini MA; Schuchman EH
    Mol Ther; 2006 Mar; 13(3):556-64. PubMed ID: 16214420
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease.
    Ziegler RJ; Brown C; Barbon CM; D'Angona AM; Schuchman EH; Andrews L; Thurberg BL; McPherson JM; Karey KP; Cheng SH
    Mol Genet Metab; 2009 May; 97(1):35-42. PubMed ID: 19231265
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion.
    Ziegler RJ; Salegio EA; Dodge JC; Bringas J; Treleaven CM; Bercury SD; Tamsett TJ; Shihabuddin L; Hadaczek P; Fiandaca M; Bankiewicz K; Scheule RK
    Exp Neurol; 2011 Oct; 231(2):261-71. PubMed ID: 21777586
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency:the utility of animal models of disease in the toxicological evaluation of potential therapeutics.
    Murray JM; Thompson AM; Vitsky A; Hawes M; Chuang WL; Pacheco J; Wilson S; McPherson JM; Thurberg BL; Karey KP; Andrews L
    Mol Genet Metab; 2015 Feb; 114(2):217-25. PubMed ID: 25092414
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Lysosomal enzyme delivery by ICAM-1-targeted nanocarriers bypassing glycosylation- and clathrin-dependent endocytosis.
    Muro S; Schuchman EH; Muzykantov VR
    Mol Ther; 2006 Jan; 13(1):135-41. PubMed ID: 16153895
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann-Pick disease.
    Pérez-Cañamás A; Benvegnù S; Rueda CB; Rábano A; Satrústegui J; Ledesma MD
    Mol Psychiatry; 2017 May; 22(5):711-723. PubMed ID: 27620840
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Sphingomyelin 16:0 is a therapeutic target for neuronal death in acid sphingomyelinase deficiency.
    Gaudioso Á; Jiang X; Casas J; Schuchman EH; Ledesma MD
    Cell Death Dis; 2023 Apr; 14(4):248. PubMed ID: 37024473
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Lipid-induced lysosomal damage after demyelination corrupts microglia protective function in lysosomal storage disorders.
    Gabandé-Rodríguez E; Pérez-Cañamás A; Soto-Huelin B; Mitroi DN; Sánchez-Redondo S; Martínez-Sáez E; Venero C; Peinado H; Ledesma MD
    EMBO J; 2019 Jan; 38(2):. PubMed ID: 30530526
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Induced Pluripotent Stem Cells for Disease Modeling and Evaluation of Therapeutics for Niemann-Pick Disease Type A.
    Long Y; Xu M; Li R; Dai S; Beers J; Chen G; Soheilian F; Baxa U; Wang M; Marugan JJ; Muro S; Li Z; Brady R; Zheng W
    Stem Cells Transl Med; 2016 Dec; 5(12):1644-1655. PubMed ID: 27484861
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Improvement of biochemical and behavioral defects in the Niemann-Pick type A mouse by intraventricular infusion of MARCKS.
    Trovò L; Stroobants S; D'Hooge R; Ledesma MD; Dotti CG
    Neurobiol Dis; 2015 Jan; 73():319-26. PubMed ID: 25251606
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery.
    Treleaven CM; Tamsett T; Fidler JA; Taksir TV; Cheng SH; Shihabuddin LS; Dodge JC
    PLoS One; 2011 Jan; 6(1):e16313. PubMed ID: 21283548
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.