BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

254 related articles for article (PubMed ID: 36631545)

  • 1. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease.
    Jeyakumar JM; Kia A; Tam LCS; McIntosh J; Spiewak J; Mills K; Heywood W; Chisari E; Castaldo N; Verhoef D; Hosseini P; Kalcheva P; Cocita C; Miranda CJ; Canavese M; Khinder J; Rosales C; Hughes D; Sheridan R; Corbau R; Nathwani A
    Gene Ther; 2023 Jun; 30(6):487-502. PubMed ID: 36631545
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. 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]  

  • 4. Systematic gene therapy derived from an investigative study of AAV2/8 vector gene therapy for Fabry disease.
    Deng M; Zhou H; He S; Qiu H; Wang Y; Zhao AY; Mu Y; Li F; Zhao AZ
    Orphanet J Rare Dis; 2023 Sep; 18(1):275. PubMed ID: 37670350
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. 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]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
    Yasuda M; Huston MW; Pagant S; Gan L; St Martin S; Sproul S; Richards D; Ballaron S; Hettini K; Ledeboer A; Falese L; Cao L; Lu Y; Holmes MC; Meyer K; Desnick RJ; Wechsler T
    Mol Ther Methods Clin Dev; 2020 Sep; 18():607-619. PubMed ID: 32775495
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. Distributions of Globotriaosylceramide Isoforms, and Globotriaosylsphingosine and Its Analogues in an α-Galactosidase A Knockout Mouse, a Model of Fabry Disease.
    Sueoka H; Aoki M; Tsukimura T; Togawa T; Sakuraba H
    PLoS One; 2015; 10(12):e0144958. PubMed ID: 26661087
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Transgenic mouse expressing human mutant alpha-galactosidase A in an endogenous enzyme deficient background: a biochemical animal model for studying active-site specific chaperone therapy for Fabry disease.
    Ishii S; Yoshioka H; Mannen K; Kulkarni AB; Fan JQ
    Biochim Biophys Acta; 2004 Nov; 1690(3):250-7. PubMed ID: 15511632
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. 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]  

  • 16. Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients.
    Young-Gqamana B; Brignol N; Chang HH; Khanna R; Soska R; Fuller M; Sitaraman SA; Germain DP; Giugliani R; Hughes DA; Mehta A; Nicholls K; Boudes P; Lockhart DJ; Valenzano KJ; Benjamin ER
    PLoS One; 2013; 8(3):e57631. PubMed ID: 23472096
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. A comprehensive testing algorithm for the diagnosis of Fabry disease in males and females.
    Stiles AR; Zhang H; Dai J; McCaw P; Beasley J; Rehder C; Koeberl DD; McDonald M; Bali DS; Young SP
    Mol Genet Metab; 2020 Jul; 130(3):209-214. PubMed ID: 32418857
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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]  

  • 20. 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; 38(5):3145-52. PubMed ID: 20131008
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.