BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

235 related articles for article (PubMed ID: 25418946)

  • 1. Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery.
    McIntyre C; Derrick-Roberts AL; Byers S; Anson DS
    J Gene Med; 2014; 16(11-12):374-87. PubMed ID: 25418946
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA.
    McIntyre C; Derrick Roberts AL; Ranieri E; Clements PR; Byers S; Anson DS
    Mol Genet Metab; 2008 Apr; 93(4):411-8. PubMed ID: 18248829
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral-mediated gene transfer.
    McIntyre C; Byers S; Anson DS
    J Gene Med; 2010 Sep; 12(9):717-28. PubMed ID: 20683858
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII.
    Bielicki J; McIntyre C; Anson DS
    Mol Genet Metab; 2010 Dec; 101(4):370-82. PubMed ID: 20864369
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA.
    King B; Hassiotis S; Rozaklis T; Beard H; Trim PJ; Snel MF; Hopwood JJ; Hemsley KM
    J Neurochem; 2016 May; 137(3):409-22. PubMed ID: 26762778
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes.
    Fraldi A; Hemsley K; Crawley A; Lombardi A; Lau A; Sutherland L; Auricchio A; Ballabio A; Hopwood JJ
    Hum Mol Genet; 2007 Nov; 16(22):2693-702. PubMed ID: 17725987
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery.
    Fu H; DiRosario J; Kang L; Muenzer J; McCarty DM
    J Gene Med; 2010 Jul; 12(7):624-33. PubMed ID: 20603889
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice.
    Duncan FJ; Naughton BJ; Zaraspe K; Murrey DA; Meadows AS; Clark KR; Newsom DE; White P; Fu H; McCarty DM
    Mol Ther; 2015 Apr; 23(4):638-47. PubMed ID: 25592334
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Characterization of a C57BL/6 congenic mouse strain of mucopolysaccharidosis type IIIA.
    Crawley AC; Gliddon BL; Auclair D; Brodie SL; Hirte C; King BM; Fuller M; Hemsley KM; Hopwood JJ
    Brain Res; 2006 Aug; 1104(1):1-17. PubMed ID: 16828069
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Brain Targeting in MPS-IIIA.
    Sorrentino NC; Fraldi A
    Pediatr Endocrinol Rev; 2016 Jun; 13 Suppl 1():630-8. PubMed ID: 27491210
    [TBL] [Abstract][Full Text] [Related]  

  • 11. A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome.
    Winner LK; Beard H; Hassiotis S; Lau AA; Luck AJ; Hopwood JJ; Hemsley KM
    Hum Gene Ther; 2016 May; 27(5):363-75. PubMed ID: 26975339
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Low-dose, continual enzyme delivery ameliorates some aspects of established brain disease in a mouse model of a childhood-onset neurodegenerative disorder.
    King B; Setford ML; Hassiotis S; Trim PJ; Duplock S; Tucker JN; Hattersley K; Snel MF; Hopwood JJ; Hemsley KM
    Exp Neurol; 2016 Apr; 278():11-21. PubMed ID: 26626972
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice.
    Gliddon BL; Hopwood JJ
    Pediatr Res; 2004 Jul; 56(1):65-72. PubMed ID: 15128919
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs--a proof of principle study.
    Hemsley KM; Norman EJ; Crawley AC; Auclair D; King B; Fuller M; Lang DL; Dean CJ; Jolly RD; Hopwood JJ
    Mol Genet Metab; 2009 Dec; 98(4):383-92. PubMed ID: 19699666
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Comparative analysis of brain pathology in heparan sulphate storing mucopolysaccharidoses.
    Derrick-Roberts A; Kaidonis X; Jackson MR; Liaw WC; Ding X; Ong C; Ranieri E; Sharp P; Fletcher J; Byers S
    Mol Genet Metab; 2020; 131(1-2):197-205. PubMed ID: 32739280
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.
    Cressant A; Desmaris N; Verot L; Bréjot T; Froissart R; Vanier MT; Maire I; Heard JM
    J Neurosci; 2004 Nov; 24(45):10229-39. PubMed ID: 15537895
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer.
    Villani GR; Follenzi A; Vanacore B; Di Domenico C; Naldini L; Di Natale P
    Biochem J; 2002 Jun; 364(Pt 3):747-53. PubMed ID: 12049639
    [TBL] [Abstract][Full Text] [Related]  

  • 18. High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector.
    Quiviger M; Arfi A; Mansard D; Delacotte L; Pastor M; Scherman D; Marie C
    Gene Ther; 2014 Dec; 21(12):1001-7. PubMed ID: 25142140
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA.
    Anson DS; McIntyre C; Thomas B; Koldej R; Ranieri E; Roberts A; Clements PR; Dunning K; Byers S
    Genet Vaccines Ther; 2007 Jan; 5():1. PubMed ID: 17227588
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA.
    Roberts AL; Rees MH; Klebe S; Fletcher JM; Byers S
    Mol Genet Metab; 2007; 92(1-2):115-21. PubMed ID: 17681480
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 12.