BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

150 related articles for article (PubMed ID: 17191071)

  • 1. Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A.
    Ziegler RJ; Cherry M; Barbon CM; Li C; Bercury SD; Armentano D; Desnick RJ; Cheng SH
    Mol Ther; 2007 Mar; 15(3):492-500. PubMed ID: 17191071
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8-mediated Hepatic Expression of α-galactosidase A.
    Ziegler RJ; Cherry M; Barbon CM; Li C; Bercury SD; Armentano D; Desnick RJ; Cheng SH
    Mol Ther; 2007 Mar; 15(3):492-500. PubMed ID: 28182896
    [TBL] [Abstract][Full Text] [Related]  

  • 3. AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.
    Ziegler RJ; Lonning SM; Armentano D; Li C; Souza DW; Cherry M; Ford C; Barbon CM; Desnick RJ; Gao G; Wilson JM; Peluso R; Godwin S; Carter BJ; Gregory RJ; Wadsworth SC; Cheng SH
    Mol Ther; 2004 Feb; 9(2):231-40. PubMed ID: 14759807
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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; 98(5):2676-81. PubMed ID: 11226298
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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; 96(3):91-6. PubMed ID: 19091614
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease.
    Ziegler RJ; Li C; Cherry M; Zhu Y; Hempel D; van Rooijen N; Ioannou YA; Desnick RJ; Goldberg MA; Yew NS; Cheng SH
    Hum Gene Ther; 2002 May; 13(8):935-45. PubMed ID: 12031126
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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; 99(21):13777-82. PubMed ID: 12370426
    [TBL] [Abstract][Full Text] [Related]  

  • 8. 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; 10(10):1667-82. PubMed ID: 10428212
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer.
    Park J; Murray GJ; Limaye A; Quirk JM; Gelderman MP; Brady RO; Qasba P
    Proc Natl Acad Sci U S A; 2003 Mar; 100(6):3450-4. PubMed ID: 12624185
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression.
    Nietupski JB; Hurlbut GD; Ziegler RJ; Chu Q; Hodges BL; Ashe KM; Bree M; Cheng SH; Gregory RJ; Marshall J; Scheule RK
    Mol Ther; 2011 Nov; 19(11):1999-2011. PubMed ID: 21712814
    [TBL] [Abstract][Full Text] [Related]  

  • 11. α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine trafficking.
    Passineau MJ; Fahrenholz T; Machen L; Zourelias L; Nega K; Paul R; MacDougall MJ; Mamaeva O; Steet R; Barnes J; Kingston HM; Benza RL
    Hum Gene Ther; 2011 Mar; 22(3):293-301. PubMed ID: 20858137
    [TBL] [Abstract][Full Text] [Related]  

  • 12. 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; 70(11):1503-8. PubMed ID: 17062978
    [TBL] [Abstract][Full Text] [Related]  

  • 13. 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; 6(1):85-92. PubMed ID: 14716680
    [TBL] [Abstract][Full Text] [Related]  

  • 14. ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice.
    Pagant S; Huston MW; Moreira L; Gan L; St Martin S; Sproul S; Holmes MC; Meyer K; Wechsler T; Desnick RJ; Yasuda M
    Mol Ther; 2021 Nov; 29(11):3230-3242. PubMed ID: 33775910
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer.
    Choi JO; Lee MH; Park HY; Jung SC
    J Biomed Sci; 2010 Apr; 17(1):26. PubMed ID: 20398385
    [TBL] [Abstract][Full Text] [Related]  

  • 16. 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; 14(3):256-65. PubMed ID: 16929352
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease.
    Li C; Ziegler RJ; Cherry M; Lukason M; Desnick RJ; Yew NS; Cheng SH
    Mol Ther; 2002 Jun; 5(6):745-54. PubMed ID: 12027559
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Purification and characterization of human alpha-galactosidase A expressed in insect cells using a baculovirus vector.
    Chen Y; Jin M; Goodrich L; Smith G; Coppola G; Calhoun DH
    Protein Expr Purif; 2000 Nov; 20(2):228-36. PubMed ID: 11049747
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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; 12(2):264-73. PubMed ID: 15946902
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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; 98(6):3428-33. PubMed ID: 11248095
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 8.