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Journal Abstract Search

697 related items for PubMed ID: 30297904

  • 1. Treatment of a metabolic liver disease by in vivo genome base editing in adult mice.
    Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Häberle J, Matos J, Robinson MD, Thöny B, Schwank G.
    Nat Med; 2018 Oct; 24(10):1519-1525. PubMed ID: 30297904
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  • 5. State-of-the-Art 2019 on Gene Therapy for Phenylketonuria.
    Grisch-Chan HM, Schwank G, Harding CO, Thöny B.
    Hum Gene Ther; 2019 Oct; 30(10):1274-1283. PubMed ID: 31364419
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  • 7. Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system.
    Ding Z, Harding CO, Rebuffat A, Elzaouk L, Wolff JA, Thöny B.
    Mol Ther; 2008 Apr; 16(4):673-81. PubMed ID: 18362925
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  • 8. In vivo prime editing of a metabolic liver disease in mice.
    Böck D, Rothgangl T, Villiger L, Schmidheini L, Matsushita M, Mathis N, Ioannidi E, Rimann N, Grisch-Chan HM, Kreutzer S, Kontarakis Z, Kopf M, Thöny B, Schwank G.
    Sci Transl Med; 2022 Mar 16; 14(636):eabl9238. PubMed ID: 35294257
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  • 10. Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer.
    Oh HJ, Park ES, Kang S, Jo I, Jung SC.
    Pediatr Res; 2004 Aug 16; 56(2):278-84. PubMed ID: 15181195
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  • 11. 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 16; 13(7):587-93. PubMed ID: 16319947
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  • 15. A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant.
    Brooks DL, Whittaker MN, Said H, Dwivedi G, Qu P, Musunuru K, Ahrens-Nicklas RC, Alameh MG, Wang X.
    HGG Adv; 2024 Jan 11; 5(1):100253. PubMed ID: 37922902
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  • 18. A bone mineralization defect in the Pahenu2 model of classical phenylketonuria involves compromised mesenchymal stem cell differentiation.
    Dobrowolski SF, Tourkova IL, Robinson LJ, Secunda C, Spridik K, Blair HC.
    Mol Genet Metab; 2018 Nov 11; 125(3):193-199. PubMed ID: 30201326
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  • 19. DNA methylation in the pathophysiology of hyperphenylalaninemia in the PAH(enu2) mouse model of phenylketonuria.
    Dobrowolski SF, Lyons-Weiler J, Spridik K, Vockley J, Skvorak K, Biery A.
    Mol Genet Metab; 2016 Sep 11; 119(1-2):1-7. PubMed ID: 26822703
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