BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

190 related articles for article (PubMed ID: 32335981)

  • 1. Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector.
    Beegle J; Hendrix K; Maciel H; Nolta JA; Anderson JS
    J Gene Med; 2020 Sep; 22(9):e3205. PubMed ID: 32335981
    [TBL] [Abstract][Full Text] [Related]  

  • 2. [Molecular pathogenesis and therapeutic approach of GM2 gangliosidosis].
    Tsuji D
    Yakugaku Zasshi; 2013; 133(2):269-74. PubMed ID: 23370522
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis.
    Ornaghi F; Sala D; Tedeschi F; Maffia MC; Bazzucchi M; Morena F; Valsecchi M; Aureli M; Martino S; Gritti A
    Neurobiol Dis; 2020 Feb; 134():104667. PubMed ID: 31682993
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Investigating Immune Responses to the scAAV9-
    Kot S; Karumuthil-Melethil S; Woodley E; Zaric V; Thompson P; Chen Z; Lykken E; Keimel JG; Kaemmerer WF; Gray SJ; Walia JS
    Int J Mol Sci; 2021 Jun; 22(13):. PubMed ID: 34201771
    [TBL] [Abstract][Full Text] [Related]  

  • 5. A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases.
    Ou L; Przybilla MJ; Tăbăran AF; Overn P; O'Sullivan MG; Jiang X; Sidhu R; Kell PJ; Ory DS; Whitley CB
    Gene Ther; 2020 May; 27(5):226-236. PubMed ID: 31896760
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Apoptotic cell death in mouse models of GM2 gangliosidosis and observations on human Tay-Sachs and Sandhoff diseases.
    Huang JQ; Trasler JM; Igdoura S; Michaud J; Hanal N; Gravel RA
    Hum Mol Genet; 1997 Oct; 6(11):1879-85. PubMed ID: 9302266
    [TBL] [Abstract][Full Text] [Related]  

  • 7. GM2 gangliosidoses in Spain: analysis of the HEXA and HEXB genes in 34 Tay-Sachs and 14 Sandhoff patients.
    Gort L; de Olano N; Macías-Vidal J; Coll MA;
    Gene; 2012 Sep; 506(1):25-30. PubMed ID: 22789865
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Brain endothelial specific gene therapy improves experimental Sandhoff disease.
    Dogbevia G; Grasshoff H; Othman A; Penno A; Schwaninger M
    J Cereb Blood Flow Metab; 2020 Jun; 40(6):1338-1350. PubMed ID: 31357902
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.
    Lahey HG; Webber CJ; Golebiowski D; Izzo CM; Horn E; Taghian T; Rodriguez P; Batista AR; Ellis LE; Hwang M; Martin DR; Gray-Edwards H; Sena-Esteves M
    Mol Ther; 2020 Oct; 28(10):2150-2160. PubMed ID: 32592687
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Therapeutic potential of intracerebroventricular replacement of modified human β-hexosaminidase B for GM2 gangliosidosis.
    Matsuoka K; Tamura T; Tsuji D; Dohzono Y; Kitakaze K; Ohno K; Saito S; Sakuraba H; Itoh K
    Mol Ther; 2011 Jun; 19(6):1017-24. PubMed ID: 21487393
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Functionality of a bicistronic construction containing
    Shaimardanova AA; Chulpanova DS; Solovyeva VV; Aimaletdinov AM; Rizvanov AA
    Neural Regen Res; 2022 Jan; 17(1):122-129. PubMed ID: 34100447
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases.
    Phaneuf D; Wakamatsu N; Huang JQ; Borowski A; Peterson AC; Fortunato SR; Ritter G; Igdoura SA; Morales CR; Benoit G; Akerman BR; Leclerc D; Hanai N; Marth JD; Trasler JM; Gravel RA
    Hum Mol Genet; 1996 Jan; 5(1):1-14. PubMed ID: 8789434
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism.
    Sango K; Yamanaka S; Hoffmann A; Okuda Y; Grinberg A; Westphal H; McDonald MP; Crawley JN; Sandhoff K; Suzuki K; Proia RL
    Nat Genet; 1995 Oct; 11(2):170-6. PubMed ID: 7550345
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Characterization of the testis and epididymis in mouse models of human Tay Sachs and Sandhoff diseases and partial determination of accumulated gangliosides.
    Trasler J; Saberi F; Somani IH; Adamali HI; Huang JQ; Fortunato SR; Ritter G; Gu M; Aebersold R; Gravel RA; Hermo L
    Endocrinology; 1998 Jul; 139(7):3280-8. PubMed ID: 9645704
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Metabolic correction in microglia derived from Sandhoff disease model mice.
    Tsuji D; Kuroki A; Ishibashi Y; Itakura T; Itoh K
    J Neurochem; 2005 Sep; 94(6):1631-8. PubMed ID: 16092933
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Promoters for the human beta-hexosaminidase genes, HEXA and HEXB.
    Norflus F; Yamanaka S; Proia RL
    DNA Cell Biol; 1996 Feb; 15(2):89-97. PubMed ID: 8634145
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Introduction of an N-glycan sequon into HEXA enhances human beta-hexosaminidase cellular uptake in a model of Sandhoff disease.
    Matsuoka K; Tsuji D; Aikawa S; Matsuzawa F; Sakuraba H; Itoh K
    Mol Ther; 2010 Aug; 18(8):1519-26. PubMed ID: 20571546
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Characterization of inducible models of Tay-Sachs and related disease.
    Sargeant TJ; Drage DJ; Wang S; Apostolakis AA; Cox TM; Cachón-González MB
    PLoS Genet; 2012 Sep; 8(9):e1002943. PubMed ID: 23028353
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Knock-down of HEXA and HEXB genes correlate with the absence of the immunostimulatory function of HSC-derived dendritic cells.
    Tiribuzi R; D'Angelo F; Berardi AC; Martino S; Orlacchio A
    Cell Biochem Funct; 2012 Jan; 30(1):61-8. PubMed ID: 21997228
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Production of recombinant beta-hexosaminidase A, a potential enzyme for replacement therapy for Tay-Sachs and Sandhoff diseases, in the methylotrophic yeast Ogataea minuta.
    Akeboshi H; Chiba Y; Kasahara Y; Takashiba M; Takaoka Y; Ohsawa M; Tajima Y; Kawashima I; Tsuji D; Itoh K; Sakuraba H; Jigami Y
    Appl Environ Microbiol; 2007 Aug; 73(15):4805-12. PubMed ID: 17557860
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 10.