151 related articles for article (PubMed ID: 12027559)
21. Enzyme replacement therapy partially prevents invariant Natural Killer T cell deficiency in the Fabry disease mouse model.
Macedo MF; Quinta R; Pereira CS; Sa Miranda MC
Mol Genet Metab; 2012 May; 106(1):83-91. PubMed ID: 22425450
[TBL] [Abstract][Full Text] [Related]
22. 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; 104(4):625-637. PubMed ID: 30879639
[TBL] [Abstract][Full Text] [Related]
23. Gene therapy for Fabry disease.
Siatskas C; Medin JA
J Inherit Metab Dis; 2001; 24 Suppl 2():25-41; discussion 11-2. PubMed ID: 11758676
[TBL] [Abstract][Full Text] [Related]
24. Enzymatic and functional correction along with long-term enzyme secretion from transduced bone marrow hematopoietic stem/progenitor and stromal cells derived from patients with Fabry disease.
Takenaka T; Hendrickson CS; Tworek DM; Tudor M; Schiffmann R; Brady RO; Medin JA
Exp Hematol; 1999 Jul; 27(7):1149-59. PubMed ID: 10390190
[TBL] [Abstract][Full Text] [Related]
25. 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
[TBL] [Abstract][Full Text] [Related]
26. Expression of genes and their responses to enzyme replacement therapy in a Fabry disease mouse model.
Park ES; Choi JO; Park JW; Lee MH; Park HY; Jung SC
Int J Mol Med; 2009 Sep; 24(3):401-7. PubMed ID: 19639234
[TBL] [Abstract][Full Text] [Related]
27. [Fabry's disease (alpha-galactosidase-A deficiency): recent therapeutic innovations].
Germain DP
J Soc Biol; 2002; 196(2):183-90. PubMed ID: 12360747
[TBL] [Abstract][Full Text] [Related]
28. 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; 11(4):325-9. PubMed ID: 12162373
[TBL] [Abstract][Full Text] [Related]
29. Characterization of a chemically modified plant cell culture expressed human α-Galactosidase-A enzyme for treatment of Fabry disease.
Kizhner T; Azulay Y; Hainrichson M; Tekoah Y; Arvatz G; Shulman A; Ruderfer I; Aviezer D; Shaaltiel Y
Mol Genet Metab; 2015 Feb; 114(2):259-67. PubMed ID: 25155442
[TBL] [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; 29(11):3230-3242. PubMed ID: 33775910
[TBL] [Abstract][Full Text] [Related]
31. 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; 16(5-6):216-21. PubMed ID: 20454522
[TBL] [Abstract][Full Text] [Related]
32. Retroviral coexpression of a multidrug resistance gene (MDR1) and human alpha-galactosidase A for gene therapy of Fabry disease.
Sugimoto Y; Aksentijevich I; Murray GJ; Brady RO; Pastan I; Gottesman MM
Hum Gene Ther; 1995 Jul; 6(7):905-15. PubMed ID: 7578409
[TBL] [Abstract][Full Text] [Related]
33. 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]
34. Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model.
Hayashi Y; Sehara Y; Watano R; Ohba K; Takayanagi Y; Sakiyama Y; Muramatsu K; Mizukami H
Hum Gene Ther; 2024 Mar; 35(5-6):192-201. PubMed ID: 38386497
[TBL] [Abstract][Full Text] [Related]
35. Reduced glucosylceramide in the mouse model of Fabry disease: correction by successful enzyme replacement therapy.
Quinta R; Rodrigues D; Assunção M; Macedo MF; Azevedo O; Cunha D; Oliveira P; Sá Miranda MC
Gene; 2014 Feb; 536(1):97-104. PubMed ID: 24334116
[TBL] [Abstract][Full Text] [Related]
36. Therapeutic strategy for Fabry disease by intravenous administration of adeno-associated virus 2 or 9 in α-galactosidase A-deficient mice.
Hayashi Y; Sehara Y; Watano R; Ohba K; Takayanagi Y; Muramatsu K; Sakiyama Y; Mizukami H
J Gene Med; 2023 Dec; 25(12):e3560. PubMed ID: 37392007
[TBL] [Abstract][Full Text] [Related]
37. [Fabry disease].
Jakubowska E; Ryba M; Hruby Z
Przegl Lek; 2006; 63(4):218-9. PubMed ID: 17080745
[TBL] [Abstract][Full Text] [Related]
38. Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease.
Schiffmann R; Murray GJ; Treco D; Daniel P; Sellos-Moura M; Myers M; Quirk JM; Zirzow GC; Borowski M; Loveday K; Anderson T; Gillespie F; Oliver KL; Jeffries NO; Doo E; Liang TJ; Kreps C; Gunter K; Frei K; Crutchfield K; Selden RF; Brady RO
Proc Natl Acad Sci U S A; 2000 Jan; 97(1):365-70. PubMed ID: 10618424
[TBL] [Abstract][Full Text] [Related]
39. Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice.
Ioannou YA; Zeidner KM; Gordon RE; Desnick RJ
Am J Hum Genet; 2001 Jan; 68(1):14-25. PubMed ID: 11115376
[TBL] [Abstract][Full Text] [Related]
40. Retrovirus-mediated transfer of human alpha-galactosidase A gene to human CD34+ hematopoietic progenitor cells.
Takiyama N; Dunigan JT; Vallor MJ; Kase R; Sakuraba H; Barranger JA
Hum Gene Ther; 1999 Dec; 10(18):2881-9. PubMed ID: 10609650
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
