BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

251 related articles for article (PubMed ID: 21917493)

  • 1. Hepatocytes from wild-type or heterozygous donors are equally effective in achieving successful therapeutic liver repopulation in murine phenylketonuria (PKU).
    Hamman KJ; Winn SR; Harding CO
    Mol Genet Metab; 2011 Nov; 104(3):235-40. PubMed ID: 21917493
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Low therapeutic threshold for hepatocyte replacement in murine phenylketonuria.
    Hamman K; Clark H; Montini E; Al-Dhalimy M; Grompe M; Finegold M; Harding CO
    Mol Ther; 2005 Aug; 12(2):337-44. PubMed ID: 16043102
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Therapeutic liver repopulation for phenylketonuria.
    Harding CO; Gibson KM
    J Inherit Metab Dis; 2010 Dec; 33(6):681-7. PubMed ID: 20495959
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.
    Harding CO; Gillingham MB; Hamman K; Clark H; Goebel-Daghighi E; Bird A; Koeberl DD
    Gene Ther; 2006 Mar; 13(5):457-62. PubMed ID: 16319949
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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; 119(1-2):1-7. PubMed ID: 26822703
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pah(enu2) mice.
    Harding CO; Neff M; Jones K; Wild K; Wolff JA
    J Gene Med; 2003 Nov; 5(11):984-93. PubMed ID: 14601136
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Blood phenylalanine reduction reverses gene expression changes observed in a mouse model of phenylketonuria.
    Manek R; Zhang YV; Berthelette P; Hossain M; Cornell CS; Gans J; Anarat-Cappillino G; Geller S; Jackson R; Yu D; Singh K; Ryan S; Bangari DS; Xu EY; Kyostio-Moore SRM
    Sci Rep; 2021 Nov; 11(1):22886. PubMed ID: 34819582
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice.
    Mochizuki S; Mizukami H; Ogura T; Kure S; Ichinohe A; Kojima K; Matsubara Y; Kobayahi E; Okada T; Hoshika A; Ozawa K; Kume A
    Gene Ther; 2004 Jul; 11(13):1081-6. PubMed ID: 15057263
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Metabolic basis of sexual dimorphism in PKU mice after genome-targeted PAH gene therapy.
    Chen L; Thung SN; Woo SL
    Mol Ther; 2007 Jun; 15(6):1079-85. PubMed ID: 17406346
    [TBL] [Abstract][Full Text] [Related]  

  • 11. A bone mineralization defect in the Pah
    Dobrowolski SF; Tourkova IL; Robinson LJ; Secunda C; Spridik K; Blair HC
    Mol Genet Metab; 2018 Nov; 125(3):193-199. PubMed ID: 30201326
    [TBL] [Abstract][Full Text] [Related]  

  • 12. A novel Pah-exon1 deleted murine model of phenylalanine hydroxylase (PAH) deficiency.
    Richards DY; Winn SR; Dudley S; Fedorov L; Rimann N; Thöny B; Harding CO
    Mol Genet Metab; 2020 Nov; 131(3):306-315. PubMed ID: 33051130
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Comparative metabolomics in the Pah
    Dobrowolski SF; Phua YL; Sudano C; Spridik K; Zinn PO; Wang Y; Bharathi S; Vockley J; Goetzman E
    Mol Genet Metab; 2022 May; 136(1):38-45. PubMed ID: 35367142
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. Comparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuria.
    Rebuffat A; Harding CO; Ding Z; Thöny B
    Hum Gene Ther; 2010 Apr; 21(4):463-77. PubMed ID: 19916803
    [TBL] [Abstract][Full Text] [Related]  

  • 16. 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; 56(2):278-84. PubMed ID: 15181195
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Mesenchymal stem cell energy deficit and oxidative stress contribute to osteopenia in the Pah
    Dobrowolski SF; Sudano C; Phua YL; Tourkova IL; Spridik K; Goetzman ES; Vockley J; Blair HC
    Mol Genet Metab; 2021 Mar; 132(3):173-179. PubMed ID: 33602601
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 19. The S-oxidation of S-carboxymethyl-L-cysteine in hepatic cytosolic fractions from BTBR and phenylketonuria enu1 and enu2 mice.
    Steventon GB; Mitchell SC
    Xenobiotica; 2019 Apr; 49(4):495-502. PubMed ID: 29648495
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Blood phenylalanine reduction corrects CNS dopamine and serotonin deficiencies and partially improves behavioral performance in adult phenylketonuric mice.
    Winn SR; Scherer T; Thöny B; Ying M; Martinez A; Weber S; Raber J; Harding CO
    Mol Genet Metab; 2018 Jan; 123(1):6-20. PubMed ID: 29331172
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.