Long-term effects of enzyme replacement therapy on Fabry cardiomy-opathy: Evidence for a better outcome with early treatment
Weidemann F, et al. (2009) Long-term effects of enzyme replacement therapy on Fabry cardiomy-opathy: evidence for a better outcome with early treatment. Circulation. 119:524-9
Correction in trans for Fabry disease: Expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vec- tor
Medin JA, et al. (1996) Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vec- tor. Proc. Natl. Acad. Sci. U. S. A. 93:7917-22
Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells
Takenaka T, et al. (2000) Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells. Proc. Natl. Acad. Sci. U. S. A. 97:7515-20
Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors
Yoshimitsu M, et al. (2004) Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors. Proc. Natl. Acad. Sci. U. S. A. 101:16909-14
Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/ progenitor cells
Yoshimitsu M, et al. (2007) Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/ progenitor cells. Gene Ther. 14:256-65
The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors
Xia H, Mao Q, Davidson BL. (2001) The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors. Nat. Biotechnol. 19:640-4
Improved intracellular delivery of glucocerebrosidase mediated by the HIV-1 TAT protein transduction domain
Lee KO, et al. (2005) Improved intracellular delivery of glucocerebrosidase mediated by the HIV-1 TAT protein transduction domain. Biochem. Bio-phys. Res. Commun. 337:701-7
A truncated HIV-1 Tat protein basic domain rapidly translo- cates through the plasma membrane and accumulates in the cell nucleus
Vives E, Brodin P, Lebleu B. (1997) A truncated HIV-1 Tat protein basic domain rapidly translo- cates through the plasma membrane and accumulates in the cell nucleus. J. Biol. Chem. 272:16010-7