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157 related items for PubMed ID: 20846825
21. Enzymatic corrections for cells derived from Fabry disease patients by a recombinant adenovirus vector. Ohsugi K, Kobayashi K, Itoh K, Sakuraba H, Sakuragawa N. J Hum Genet; 2000; 45(1):1-5. PubMed ID: 10697955 [Abstract] [Full Text] [Related]
24. Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction. Kyosen SO, Iizuka S, Kobayashi H, Kimura T, Fukuda T, Shen J, Shimada Y, Ida H, Eto Y, Ohashi T. Gene Ther; 2010 Apr; 17(4):521-30. PubMed ID: 20033064 [Abstract] [Full Text] [Related]
29. HIV-2 derived lentiviral vectors: gene transfer in Parkinson's and Fabry disease models in vitro. D'Costa J, Harvey-White J, Qasba P, Limaye A, Kaneski CR, Davis-Warren A, Brady RO, Bankiewicz KS, Major EO, Arya SK. J Med Virol; 2003 Oct 16; 71(2):173-82. PubMed ID: 12938190 [Abstract] [Full Text] [Related]
30. 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 03; 29(11):3230-3242. PubMed ID: 33775910 [Abstract] [Full Text] [Related]
31. Improved lentiviral vectors for Wiskott-Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis. Frecha C, Toscano MG, Costa C, Saez-Lara MJ, Cosset FL, Verhoeyen E, Martin F. Gene Ther; 2008 Jun 03; 15(12):930-41. PubMed ID: 18323794 [Abstract] [Full Text] [Related]
32. An easy and sensitive method for determination of globotriaosylceramide (Gb3) from urinary sediment: utility for Fabry disease diagnosis and treatment monitoring. Rozenfeld PA, De Francesco NP, Borrajo GJ, Ceci R, Fossati CA. Clin Chim Acta; 2009 May 03; 403(1-2):194-7. PubMed ID: 19268437 [Abstract] [Full Text] [Related]
33. 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 20; 13(8):935-45. PubMed ID: 12031126 [Abstract] [Full Text] [Related]
34. Sequencing and characterization of the porcine α-galactosidase A gene: towards the generation of a porcine model for Fabry disease. Yoshimitsu M, Higuchi K, Fan X, Takao S, Medin JA, Tei C, Takenaka T. Mol Biol Rep; 2011 Jun 20; 38(5):3145-52. PubMed ID: 20131008 [Abstract] [Full Text] [Related]
35. Lentiviral vectors for delivery of genes into neonatal and adult ventricular cardiac myocytes in vitro and in vivo. Zhao J, Pettigrew GJ, Thomas J, Vandenberg JI, Delriviere L, Bolton EM, Carmichael A, Martin JL, Marber MS, Lever AM. Basic Res Cardiol; 2002 Sep 20; 97(5):348-58. PubMed ID: 12200634 [Abstract] [Full Text] [Related]
36. Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates. Zhu X, Yin L, Theisen M, Zhuo J, Siddiqui S, Levy B, Presnyak V, Frassetto A, Milton J, Salerno T, Benenato KE, Milano J, Lynn A, Sabnis S, Burke K, Besin G, Lukacs CM, Guey LT, Finn PF, Martini PGV. Am J Hum Genet; 2019 Apr 04; 104(4):625-637. PubMed ID: 30879639 [Abstract] [Full Text] [Related]