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Journal Abstract Search

512 related items for PubMed ID: 29764210

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  • 2. Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1.
    Hickey RD, Mao SA, Glorioso J, Elgilani F, Amiot B, Chen H, Rinaldo P, Marler R, Jiang H, DeGrado TR, Suksanpaisan L, O'Connor MK, Freeman BL, Ibrahim SH, Peng KW, Harding CO, Ho CS, Grompe M, Ikeda Y, Lillegard JB, Russell SJ, Nyberg SL.
    Sci Transl Med; 2016 Jul 27; 8(349):349ra99. PubMed ID: 27464750
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  • 3. Cas9-nickase-mediated genome editing corrects hereditary tyrosinemia in rats.
    Shao Y, Wang L, Guo N, Wang S, Yang L, Li Y, Wang M, Yin S, Han H, Zeng L, Zhang L, Hui L, Ding Q, Zhang J, Geng H, Liu M, Li D.
    J Biol Chem; 2018 May 04; 293(18):6883-6892. PubMed ID: 29507093
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  • 4. Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1.
    Hickey RD, Nicolas CT, Allen K, Mao S, Elgilani F, Glorioso J, Amiot B, VanLith C, Guthman R, Du Z, Chen H, Harding CO, Kaiser RA, Nyberg SL, Lillegard JB.
    Cell Transplant; 2019 Jan 04; 28(1):79-88. PubMed ID: 30477316
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  • 8. CRISPR/Cas9-Mediated Gene Correction in Newborn Rabbits with Hereditary Tyrosinemia Type I.
    Li N, Gou S, Wang J, Zhang Q, Huang X, Xie J, Li L, Jin Q, Ouyang Z, Chen F, Ge W, Shi H, Liang Y, Zhuang Z, Zhao X, Lian M, Ye Y, Quan L, Wu H, Lai L, Wang K.
    Mol Ther; 2021 Mar 03; 29(3):1001-1015. PubMed ID: 33221434
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  • 11. High volume naked DNA tail-vein injection restores liver function in Fah-knock out mice.
    Eggenhofer E, Doenecke A, Renner P, Slowik P, Piso P, Geissler EK, Schlitt HJ, Dahlke MH, Popp FC.
    J Gastroenterol Hepatol; 2010 May 03; 25(5):1002-8. PubMed ID: 20546455
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  • 13. CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.
    Ohmori T, Nagao Y, Mizukami H, Sakata A, Muramatsu SI, Ozawa K, Tominaga SI, Hanazono Y, Nishimura S, Nureki O, Sakata Y.
    Sci Rep; 2017 Jun 23; 7(1):4159. PubMed ID: 28646206
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  • 15. Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.
    Ibraheim R, Tai PWL, Mir A, Javeed N, Wang J, Rodríguez TC, Namkung S, Nelson S, Khokhar ES, Mintzer E, Maitland S, Chen Z, Cao Y, Tsagkaraki E, Wolfe SA, Wang D, Pai AA, Xue W, Gao G, Sontheimer EJ.
    Nat Commun; 2021 Nov 01; 12(1):6267. PubMed ID: 34725353
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  • 16. In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions.
    Nicolas CT, VanLith CJ, Hickey RD, Du Z, Hillin LG, Guthman RM, Cao WJ, Haugo B, Lillegard A, Roy D, Bhagwate A, O'Brien D, Kocher JP, Kaiser RA, Russell SJ, Lillegard JB.
    Nat Commun; 2022 Aug 25; 13(1):5012. PubMed ID: 36008405
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  • 18. Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents.
    Rittelmeyer I, Rothe M, Brugman MH, Iken M, Schambach A, Manns MP, Baum C, Modlich U, Ott M.
    Hepatology; 2013 Jul 25; 58(1):397-408. PubMed ID: 23258554
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  • 19. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.
    Yin H, Xue W, Chen S, Bogorad RL, Benedetti E, Grompe M, Koteliansky V, Sharp PA, Jacks T, Anderson DG.
    Nat Biotechnol; 2014 Jun 25; 32(6):551-3. PubMed ID: 24681508
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  • 20. Messenger RNA as a source of transposase for sleeping beauty transposon-mediated correction of hereditary tyrosinemia type I.
    Wilber A, Wangensteen KJ, Chen Y, Zhuo L, Frandsen JL, Bell JB, Chen ZJ, Ekker SC, McIvor RS, Wang X.
    Mol Ther; 2007 Jul 25; 15(7):1280-7. PubMed ID: 17440442
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