BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

313 related articles for article (PubMed ID: 34450229)

  • 1. The GM2 gangliosidoses: Unlocking the mysteries of pathogenesis and treatment.
    Toro C; Zainab M; Tifft CJ
    Neurosci Lett; 2021 Nov; 764():136195. PubMed ID: 34450229
    [No 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. 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]  

  • 5. 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]  

  • 6. 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]  

  • 7. 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]  

  • 8. GM2 gangliosidosis in Saudi Arabia: multiple mutations and considerations for future carrier screening.
    Kaya N; Al-Owain M; Abudheim N; Al-Zahrani J; Colak D; Al-Sayed M; Milanlioglu A; Ozand PT; Alkuraya FS
    Am J Med Genet A; 2011 Jun; 155A(6):1281-4. PubMed ID: 21567908
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Therapeutic evaluation of GM2 gangliosidoses by ELISA using anti-GM2 ganglioside antibodies.
    Tsuji D; Higashine Y; Matsuoka K; Sakuraba H; Itoh K
    Clin Chim Acta; 2007 Mar; 378(1-2):38-41. PubMed ID: 17196574
    [TBL] [Abstract][Full Text] [Related]  

  • 10. GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies.
    Leal AF; Benincore-Flórez E; Solano-Galarza D; Garzón Jaramillo RG; Echeverri-Peña OY; Suarez DA; Alméciga-Díaz CJ; Espejo-Mojica AJ
    Int J Mol Sci; 2020 Aug; 21(17):. PubMed ID: 32867370
    [TBL] [Abstract][Full Text] [Related]  

  • 11. CRISPR/nCas9-Based Genome Editing on GM2 Gangliosidoses Fibroblasts via Non-Viral Vectors.
    Leal AF; Cifuentes J; Quezada V; Benincore-Flórez E; Cruz JC; Reyes LH; Espejo-Mojica AJ; Alméciga-Díaz CJ
    Int J Mol Sci; 2022 Sep; 23(18):. PubMed ID: 36142595
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Highly phosphomannosylated enzyme replacement therapy for GM2 gangliosidosis.
    Tsuji D; Akeboshi H; Matsuoka K; Yasuoka H; Miyasaki E; Kasahara Y; Kawashima I; Chiba Y; Jigami Y; Taki T; Sakuraba H; Itoh K
    Ann Neurol; 2011 Apr; 69(4):691-701. PubMed ID: 21520232
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.
    Walia JS; Altaleb N; Bello A; Kruck C; LaFave MC; Varshney GK; Burgess SM; Chowdhury B; Hurlbut D; Hemming R; Kobinger GP; Triggs-Raine B
    Mol Ther; 2015 Mar; 23(3):414-22. PubMed ID: 25515709
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.
    Golebiowski D; van der Bom IMJ; Kwon CS; Miller AD; Petrosky K; Bradbury AM; Maitland S; Kühn AL; Bishop N; Curran E; Silva N; GuhaSarkar D; Westmoreland SV; Martin DR; Gounis MJ; Asaad WF; Sena-Esteves M
    Hum Gene Ther; 2017 Jun; 28(6):510-522. PubMed ID: 28132521
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Protease-resistant modified human β-hexosaminidase B ameliorates symptoms in GM2 gangliosidosis model.
    Kitakaze K; Mizutani Y; Sugiyama E; Tasaki C; Tsuji D; Maita N; Hirokawa T; Asanuma D; Kamiya M; Sato K; Setou M; Urano Y; Togawa T; Otaka A; Sakuraba H; Itoh K
    J Clin Invest; 2016 May; 126(5):1691-703. PubMed ID: 27018595
    [TBL] [Abstract][Full Text] [Related]  

  • 16. 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]  

  • 17. 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]  

  • 18. Mouse model of GM2 activator deficiency manifests cerebellar pathology and motor impairment.
    Liu Y; Hoffmann A; Grinberg A; Westphal H; McDonald MP; Miller KM; Crawley JN; Sandhoff K; Suzuki K; Proia RL
    Proc Natl Acad Sci U S A; 1997 Jul; 94(15):8138-43. PubMed ID: 9223328
    [TBL] [Abstract][Full Text] [Related]  

  • 19. The GM2 gangliosidoses databases: allelic variation at the HEXA, HEXB, and GM2A gene loci.
    Cordeiro P; Hechtman P; Kaplan F
    Genet Med; 2000; 2(6):319-27. PubMed ID: 11339652
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 16.