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Journal Abstract Search

373 related items for PubMed ID: 31402115

  • 1. Targeted gene therapy in human-induced pluripotent stem cells from a patient with primary hyperoxaluria type 1 using CRISPR/Cas9 technology.
    Estève J, Blouin JM, Lalanne M, Azzi-Martin L, Dubus P, Bidet A, Harambat J, Llanas B, Moranvillier I, Bedel A, Moreau-Gaudry F, Richard E.
    Biochem Biophys Res Commun; 2019 Oct 01; 517(4):677-683. PubMed ID: 31402115
    [Abstract] [Full Text] [Related]

  • 2. Generation of induced pluripotent stem cells-derived hepatocyte-like cells for ex vivo gene therapy of primary hyperoxaluria type 1.
    Estève J, Blouin JM, Lalanne M, Azzi-Martin L, Dubus P, Bidet A, Harambat J, Llanas B, Moranvillier I, Bedel A, Moreau-Gaudry F, Richard E.
    Stem Cell Res; 2019 Jul 01; 38():101467. PubMed ID: 31151050
    [Abstract] [Full Text] [Related]

  • 3. Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system.
    Lyu C, Shen J, Wang R, Gu H, Zhang J, Xue F, Liu X, Liu W, Fu R, Zhang L, Li H, Zhang X, Cheng T, Yang R, Zhang L.
    Stem Cell Res Ther; 2018 Apr 06; 9(1):92. PubMed ID: 29625575
    [Abstract] [Full Text] [Related]

  • 4. Generation of a Primary Hyperoxaluria Type 1 Disease Model Via CRISPR/Cas9 System in Rats.
    Zheng R, Fang X, He L, Shao Y, Guo N, Wang L, Liu M, Li D, Geng H.
    Curr Mol Med; 2018 Apr 06; 18(7):436-447. PubMed ID: 30539697
    [Abstract] [Full Text] [Related]

  • 5. CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I.
    Zabaleta N, Barberia M, Martin-Higueras C, Zapata-Linares N, Betancor I, Rodriguez S, Martinez-Turrillas R, Torella L, Vales A, Olagüe C, Vilas-Zornoza A, Castro-Labrador L, Lara-Astiaso D, Prosper F, Salido E, Gonzalez-Aseguinolaza G, Rodriguez-Madoz JR.
    Nat Commun; 2018 Dec 21; 9(1):5454. PubMed ID: 30575740
    [Abstract] [Full Text] [Related]

  • 6. Generation and characterization of a novel rat model of primary hyperoxaluria type 1 with a nonsense mutation in alanine-glyoxylate aminotransferase gene.
    Li Y, Zheng R, Xu G, Huang Y, Li Y, Li D, Geng H.
    Am J Physiol Renal Physiol; 2021 Mar 01; 320(3):F475-F484. PubMed ID: 33491567
    [Abstract] [Full Text] [Related]

  • 7. Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells.
    Klatt D, Cheng E, Hoffmann D, Santilli G, Thrasher AJ, Brendel C, Schambach A.
    Hum Gene Ther; 2020 Feb 01; 31(3-4):199-210. PubMed ID: 31773990
    [Abstract] [Full Text] [Related]

  • 8. Correction of hyperoxaluria by liver repopulation with hepatocytes in a mouse model of primary hyperoxaluria type-1.
    Jiang J, Salido EC, Guha C, Wang X, Moitra R, Liu L, Roy-Chowdhury J, Roy-Chowdhury N.
    Transplantation; 2008 May 15; 85(9):1253-60. PubMed ID: 18475180
    [Abstract] [Full Text] [Related]

  • 9. Low-density lipoprotein receptor-deficient hepatocytes differentiated from induced pluripotent stem cells allow familial hypercholesterolemia modeling, CRISPR/Cas-mediated genetic correction, and productive hepatitis C virus infection.
    Caron J, Pène V, Tolosa L, Villaret M, Luce E, Fourrier A, Heslan JM, Saheb S, Bruckert E, Gómez-Lechón MJ, Nguyen TH, Rosenberg AR, Weber A, Dubart-Kupperschmitt A.
    Stem Cell Res Ther; 2019 Jul 29; 10(1):221. PubMed ID: 31358055
    [Abstract] [Full Text] [Related]

  • 10. CRISPR/Cas9-mediated metabolic pathway reprogramming in a novel humanized rat model ameliorates primary hyperoxaluria type 1.
    Zheng R, Li Y, Wang L, Fang X, Zhang J, He L, Yang L, Li D, Geng H.
    Kidney Int; 2020 Oct 29; 98(4):947-957. PubMed ID: 32464217
    [Abstract] [Full Text] [Related]

  • 11. CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs.
    Morishige S, Mizuno S, Ozawa H, Nakamura T, Mazahery A, Nomura K, Seki R, Mouri F, Osaki K, Yamamura K, Okamura T, Nagafuji K.
    Int J Hematol; 2020 Feb 29; 111(2):225-233. PubMed ID: 31664646
    [Abstract] [Full Text] [Related]

  • 12. Systemic Alanine Glyoxylate Aminotransferase mRNA Improves Glyoxylate Metabolism in a Mouse Model of Primary Hyperoxaluria Type 1.
    Kukreja A, Lasaro M, Cobaugh C, Forbes C, Tang JP, Gao X, Martin-Higueras C, Pey AL, Salido E, Sobolov S, Subramanian RR.
    Nucleic Acid Ther; 2019 Apr 29; 29(2):104-113. PubMed ID: 30676254
    [Abstract] [Full Text] [Related]

  • 13. Generation of an induced pluripotent stem cell line (CIMAi001-A) from a compound heterozygous Primary Hyperoxaluria Type I (PH1) patient carrying p.G170R and p.R122* mutations in the AGXT gene.
    Martinez-Turrillas R, Rodriguez-Diaz S, Rodriguez-Marquez P, Martin-Mallo A, Salido E, Beck BB, Prosper F, Rodriguez-Madoz JR.
    Stem Cell Res; 2019 Dec 29; 41():101626. PubMed ID: 31715429
    [Abstract] [Full Text] [Related]

  • 14. In vivo base editing rescues primary hyperoxaluria type 1 in rats.
    Chen Z, Zhang D, Zheng R, Yang L, Huo Y, Zhang D, Fang X, Li Y, Xu G, Li D, Geng H.
    Kidney Int; 2024 Mar 29; 105(3):496-507. PubMed ID: 38142039
    [Abstract] [Full Text] [Related]

  • 15. Restored glyoxylate metabolism after AGXT gene correction and direct reprogramming of primary hyperoxaluria type 1 fibroblasts.
    Nieto-Romero V, García-Torralba A, Molinos-Vicente A, Moya FJ, Rodríguez-Perales S, García-Escudero R, Salido E, Segovia JC, García-Bravo M.
    iScience; 2024 Apr 19; 27(4):109530. PubMed ID: 38577102
    [Abstract] [Full Text] [Related]

  • 16. Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1.
    Castello R, Borzone R, D'Aria S, Annunziata P, Piccolo P, Brunetti-Pierri N.
    Gene Ther; 2016 Feb 19; 23(2):129-34. PubMed ID: 26609667
    [Abstract] [Full Text] [Related]

  • 17. Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer.
    Salido E, Rodriguez-Pena M, Santana A, Beattie SG, Petry H, Torres A.
    Mol Ther; 2011 May 19; 19(5):870-5. PubMed ID: 21119625
    [Abstract] [Full Text] [Related]

  • 18. CRISPR-Cas9-Based Genome Editing of Human Induced Pluripotent Stem Cells.
    Giacalone JC, Sharma TP, Burnight ER, Fingert JF, Mullins RF, Stone EM, Tucker BA.
    Curr Protoc Stem Cell Biol; 2018 Feb 28; 44():5B.7.1-5B.7.22. PubMed ID: 29512106
    [Abstract] [Full Text] [Related]

  • 19. Multiplex gene editing reduces oxalate production in primary hyperoxaluria type 1.
    Zheng R, Zhang DX, Shao YJ, Fang XL, Yang L, Huo YN, Li DL, Geng HQ.
    Zool Res; 2023 Nov 18; 44(6):993-1002. PubMed ID: 37759334
    [Abstract] [Full Text] [Related]

  • 20. Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1.
    VanLith C, Guthman R, Nicolas CT, Allen K, Du Z, Joo DJ, Nyberg SL, Lillegard JB, Hickey RD.
    Hum Gene Ther; 2018 Nov 18; 29(11):1315-1326. PubMed ID: 29764210
    [Abstract] [Full Text] [Related]

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