469 related articles for article (PubMed ID: 12624185)
1. 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]
2. 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]
3. 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]
4. 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]
5. 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]
6. 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]
7. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors.
Song S; Embury J; Laipis PJ; Berns KI; Crawford JM; Flotte TR
Gene Ther; 2001 Sep; 8(17):1299-306. PubMed ID: 11571566
[TBL] [Abstract][Full Text] [Related]
8. [Gene therapy of Gaucher's and Fabry's diseases: current status and prospects].
Fabrega S; Lehn P
J Soc Biol; 2002; 196(2):175-81. PubMed ID: 12360746
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. [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]
11. Fabry disease: molecular genetics of the inherited nephropathy.
Desnick RJ; Astrin KH; Bishop DF
Adv Nephrol Necker Hosp; 1989; 18():113-27. PubMed ID: 2564247
[TBL] [Abstract][Full Text] [Related]
12. Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer.
Ding Z; Georgiev P; Thöny B
Gene Ther; 2006 Apr; 13(7):587-93. PubMed ID: 16319947
[TBL] [Abstract][Full Text] [Related]
13. 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]
14. [alpha-Galactosidase gene mutation and its expression product in Fabry disease (alpha-galactosidase deficiency)].
Okumiya T; Takata T; Sasaki M; Sakuraba H
Rinsho Byori; 1997 Feb; 45(2):127-35. PubMed ID: 9120996
[TBL] [Abstract][Full Text] [Related]
15. Naked plasmid DNA-based alpha-galactosidase A gene transfer partially reduces systemic accumulation of globotriaosylceramide in Fabry mice.
Nakamura G; Maruyama H; Ishii S; Shimotori M; Kameda S; Kono T; Miyazaki J; Kulkarni AB; Gejyo F
Mol Biotechnol; 2008 Feb; 38(2):109-19. PubMed ID: 18219591
[TBL] [Abstract][Full Text] [Related]
16. Fabry disease: D313Y is an alpha-galactosidase A sequence variant that causes pseudodeficient activity in plasma.
Froissart R; Guffon N; Vanier MT; Desnick RJ; Maire I
Mol Genet Metab; 2003 Nov; 80(3):307-14. PubMed ID: 14680977
[TBL] [Abstract][Full Text] [Related]
17. Neurophysiological, behavioral and morphological abnormalities in the Fabry knockout mice.
Rodrigues LG; Ferraz MJ; Rodrigues D; Pais-Vieira M; Lima D; Brady RO; Sousa MM; Sá-Miranda MC
Neurobiol Dis; 2009 Jan; 33(1):48-56. PubMed ID: 18848893
[TBL] [Abstract][Full Text] [Related]
18. Non-viral, integrin-mediated gene transfer into fibroblasts from patients with lysosomal storage diseases.
Estruch EJ; Hart SL; Kinnon C; Winchester BG
J Gene Med; 2001; 3(5):488-97. PubMed ID: 11601762
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. Fabry disease: characterization of alpha-galactosidase A double mutations and the D313Y plasma enzyme pseudodeficiency allele.
Yasuda M; Shabbeer J; Benson SD; Maire I; Burnett RM; Desnick RJ
Hum Mutat; 2003 Dec; 22(6):486-92. PubMed ID: 14635108
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
