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Journal Abstract Search

157 related items for PubMed ID: 20846825

  • 1. Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease.
    Lee CJ, Fan X, Guo X, Medin JA.
    J Cardiol; 2011 Jan; 57(1):115-22. PubMed ID: 20846825
    [Abstract] [Full Text] [Related]

  • 2. Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer.
    Takahashi H, Hirai Y, Migita M, Seino Y, Fukuda Y, Sakuraba H, Kase R, Kobayashi T, Hashimoto Y, Shimada T.
    Proc Natl Acad Sci U S A; 2002 Oct 15; 99(21):13777-82. PubMed ID: 12370426
    [Abstract] [Full Text] [Related]

  • 3. Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.
    Yoshimitsu M, Higuchi K, Ramsubir S, Nonaka T, Rasaiah VI, Siatskas C, Liang SB, Murray GJ, Brady RO, Medin JA.
    Gene Ther; 2007 Feb 15; 14(3):256-65. PubMed ID: 16929352
    [Abstract] [Full Text] [Related]

  • 4. Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period.
    Ogawa K, Hirai Y, Ishizaki M, Takahashi H, Hanawa H, Fukunaga Y, Shimada T.
    Mol Genet Metab; 2009 Mar 15; 96(3):91-6. PubMed ID: 19091614
    [Abstract] [Full Text] [Related]

  • 5. Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.
    Przybylska M, Wu IH, Zhao H, Ziegler RJ, Tousignant JD, Desnick RJ, Scheule RK, Cheng SH, Yew NS.
    J Gene Med; 2004 Jan 15; 6(1):85-92. PubMed ID: 14716680
    [Abstract] [Full Text] [Related]

  • 6. Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice.
    Jung SC, Han IP, Limaye A, Xu R, Gelderman MP, Zerfas P, Tirumalai K, Murray GJ, During MJ, Brady RO, Qasba P.
    Proc Natl Acad Sci U S A; 2001 Feb 27; 98(5):2676-81. PubMed ID: 11226298
    [Abstract] [Full Text] [Related]

  • 7. Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells.
    Takenaka T, Murray GJ, Qin G, Quirk JM, Ohshima T, Qasba P, Clark K, Kulkarni AB, Brady RO, Medin JA.
    Proc Natl Acad Sci U S A; 2000 Jun 20; 97(13):7515-20. PubMed ID: 10840053
    [Abstract] [Full Text] [Related]

  • 8. Preselective gene therapy for Fabry disease.
    Qin G, Takenaka T, Telsch K, Kelley L, Howard T, Levade T, Deans R, Howard BH, Malech HL, Brady RO, Medin JA.
    Proc Natl Acad Sci U S A; 2001 Mar 13; 98(6):3428-33. PubMed ID: 11248095
    [Abstract] [Full Text] [Related]

  • 9. Correction of cardiac abnormalities in fabry mice by direct intraventricular injection of a recombinant lentiviral vector that engineers expression of alpha-galactosidase A.
    Yoshimitsu M, Higuchi K, Dawood F, Rasaiah VI, Ayach B, Chen M, Liu P, Medin JA.
    Circ J; 2006 Nov 13; 70(11):1503-8. PubMed ID: 17062978
    [Abstract] [Full Text] [Related]

  • 10. In vitro study of encapsulation therapy for Fabry disease using genetically engineered CHO cell line.
    Naganawa Y, Ohsugi K, Kase R, Date I, Sakuraba H, Sakuragawa N.
    Cell Transplant; 2002 Nov 13; 11(4):325-9. PubMed ID: 12162373
    [Abstract] [Full Text] [Related]

  • 11. Transient siRNA-mediated attenuation of liver expression from an alpha-galactosidase A plasmid reduces subsequent humoral immune responses to the transgene product in mice.
    Chu Q, Joseph M, Przybylska M, Yew NS, Scheule RK.
    Mol Ther; 2005 Aug 13; 12(2):264-73. PubMed ID: 15946902
    [Abstract] [Full Text] [Related]

  • 12. Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors.
    Yoshimitsu M, Sato T, Tao K, Walia JS, Rasaiah VI, Sleep GT, Murray GJ, Poeppl AG, Underwood J, West L, Brady RO, Medin JA.
    Proc Natl Acad Sci U S A; 2004 Nov 30; 101(48):16909-14. PubMed ID: 15550536
    [Abstract] [Full Text] [Related]

  • 13. Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer.
    Ziegler RJ, Yew NS, Li C, Cherry M, Berthelette P, Romanczuk H, Ioannou YA, Zeidner KM, Desnick RJ, Cheng SH.
    Hum Gene Ther; 1999 Jul 01; 10(10):1667-82. PubMed ID: 10428212
    [Abstract] [Full Text] [Related]

  • 14. Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors.
    Müller OJ, Leuchs B, Pleger ST, Grimm D, Franz WM, Katus HA, Kleinschmidt JA.
    Cardiovasc Res; 2006 Apr 01; 70(1):70-8. PubMed ID: 16448634
    [Abstract] [Full Text] [Related]

  • 15. Promoter dependence of plasmid-pluronics targeted alpha galactosidase A expression in skeletal muscle of Fabry mice.
    Lavigne MD, Pohlschmidt M, Novo JF, Higgins B, Alakhov V, Lochmuller H, Sakuraba H, Goldspink G, MacDermot K, Górecki DC.
    Mol Ther; 2005 Nov 01; 12(5):985-90. PubMed ID: 15975851
    [No Abstract] [Full Text] [Related]

  • 16. [Gene therapy of Gaucher's and Fabry's diseases: current status and prospects].
    Fabrega S, Lehn P.
    J Soc Biol; 2002 Nov 01; 196(2):175-81. PubMed ID: 12360746
    [Abstract] [Full Text] [Related]

  • 17. Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors.
    Raake PW, Hinkel R, Müller S, Delker S, Kreuzpointner R, Kupatt C, Katus HA, Kleinschmidt JA, Boekstegers P, Müller OJ.
    Gene Ther; 2008 Jan 01; 15(1):12-7. PubMed ID: 17943147
    [Abstract] [Full Text] [Related]

  • 18. High and sustained transgene expression in vivo from plasmid vectors containing a hybrid ubiquitin promoter.
    Yew NS, Przybylska M, Ziegler RJ, Liu D, Cheng SH.
    Mol Ther; 2001 Jul 01; 4(1):75-82. PubMed ID: 11472109
    [Abstract] [Full Text] [Related]

  • 19. An in vitro model of Fabry disease.
    Shu L, Murphy HS, Cooling L, Shayman JA.
    J Am Soc Nephrol; 2005 Sep 01; 16(9):2636-45. PubMed ID: 16033856
    [Abstract] [Full Text] [Related]

  • 20. Alpha-galactosidase A-Tat fusion enhances storage reduction in hearts and kidneys of Fabry mice.
    Higuchi K, Yoshimitsu M, Fan X, Guo X, Rasaiah VI, Yen J, Tei C, Takenaka T, Medin JA.
    Mol Med; 2010 Sep 01; 16(5-6):216-21. PubMed ID: 20454522
    [Abstract] [Full Text] [Related]

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