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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;15(7):1280-7. Epub 2007 Apr 17.


In vivo correction of murine tyrosinemia type I by DNA-mediated transposition.

Montini E, Held PK, Noll M, Morcinek N, Al-Dhalimy M, Finegold M, Yant SR, Kay MA, Grompe M.

Mol Ther. 2002 Dec;6(6):759-69.


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;25(5):1002-8. doi: 10.1111/j.1440-1746.2009.06156.x.


Generation of healthy mice from gene-corrected disease-specific induced pluripotent stem cells.

Wu G, Liu N, Rittelmeyer I, Sharma AD, Sgodda M, Zaehres H, Bleidissel M, Greber B, Gentile L, Han DW, Rudolph C, Steinemann D, Schambach A, Ott M, Schöler HR, Cantz T.

PLoS Biol. 2011 Jul;9(7):e1001099. doi: 10.1371/journal.pbio.1001099. Epub 2011 Jul 12.


Renal proximal tubular cells acquire resistance to cell death stimuli in mice with hereditary tyrosinemia type 1.

Luijerink MC, van Beurden EA, Malingré HE, Jacobs SM, Grompe M, Klomp LW, Berger R, van den Berg IE.

Kidney Int. 2004 Sep;66(3):990-1000.


Hereditary tyrosinaemia type I: from basics to progress in treatment.

Pitkänen ST, Salo MK, Heikinheimo M.

Ann Med. 2000 Nov;32(8):530-8. Review.


Evaluation of dichloroacetate treatment in a murine model of hereditary tyrosinemia type 1.

Langlois C, Jorquera R, Finegold M, Shroads AL, Stacpoole PW, Tanguay RM.

Biochem Pharmacol. 2006 May 28;71(11):1648-61. Epub 2006 Apr 3.


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;32(6):551-3. doi: 10.1038/nbt.2884. Epub 2014 Mar 30. Erratum in: Nat Biotechnol. 2014 Sep;32(9):952.


In vivo correction of murine hereditary tyrosinemia type I by phiC31 integrase-mediated gene delivery.

Held PK, Olivares EC, Aguilar CP, Finegold M, Calos MP, Grompe M.

Mol Ther. 2005 Mar;11(3):399-408.


Kidneys of mice with hereditary tyrosinemia type I are extremely sensitive to cytotoxicity.

Jacobs SM, van Beurden DH, Klomp LW, Berger R, van den Berg IE.

Pediatr Res. 2006 Mar;59(3):365-70.


Pharmacological rescue of the 14CoS/14CoS mouse: hepatocyte apoptosis is likely caused by endogenous oxidative stress.

Dieter MZ, Freshwater SL, Miller ML, Shertzer HG, Dalton TP, Nebert DW.

Free Radic Biol Med. 2003 Aug 15;35(4):351-67.


A facile method for somatic, lifelong manipulation of multiple genes in the mouse liver.

Wangensteen KJ, Wilber A, Keng VW, He Z, Matise I, Wangensteen L, Carson CM, Chen Y, Steer CJ, McIvor RS, Largaespada DA, Wang X, Ekker SC.

Hepatology. 2008 May;47(5):1714-24. doi: 10.1002/hep.22195.


Extensive changes in liver gene expression induced by hereditary tyrosinemia type I are not normalized by treatment with 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC).

Luijerink MC, Jacobs SM, van Beurden EA, Koornneef LP, Klomp LW, Berger R, van den Berg IE.

J Hepatol. 2003 Dec;39(6):901-9.


Point mutations in the murine fumarylacetoacetate hydrolase gene: Animal models for the human genetic disorder hereditary tyrosinemia type 1.

Aponte JL, Sega GA, Hauser LJ, Dhar MS, Withrow CM, Carpenter DA, Rinchik EM, Culiat CT, Johnson DK.

Proc Natl Acad Sci U S A. 2001 Jan 16;98(2):641-5.


Conditional gene expression in the mouse using a Sleeping Beauty gene-trap transposon.

Geurts AM, Wilber A, Carlson CM, Lobitz PD, Clark KJ, Hackett PB, McIvor RS, Largaespada DA.

BMC Biotechnol. 2006 Jun 26;6:30.


Non-viral FoxM1 gene delivery to hepatocytes enhances liver repopulation.

Xiang D, Liu CC, Wang MJ, Li JX, Chen F, Yao H, Yu B, Lu L, Borjigin U, Chen YX, Zhong L, Wangensteen KJ, He ZY, Wang X, Hu YP.

Cell Death Dis. 2014 May 22;5:e1252. doi: 10.1038/cddis.2014.230.


Rescue from neonatal death in the murine model of hereditary tyrosinemia by glutathione monoethylester and vitamin C treatment.

Langlois C, Jorquera R, Orejuela D, Bergeron A, Finegold MJ, Rhead WJ, Tanguay RM.

Mol Genet Metab. 2008 Mar;93(3):306-13. Epub 2007 Nov 26.


Chromosomal mobilization and reintegration of Sleeping Beauty and PiggyBac transposons.

Liang Q, Kong J, Stalker J, Bradley A.

Genesis. 2009 Jun;47(6):404-8. doi: 10.1002/dvg.20508.


The forkhead box M1 transcription factor contributes to the development and growth of mouse colorectal cancer.

Yoshida Y, Wang IC, Yoder HM, Davidson NO, Costa RH.

Gastroenterology. 2007 Apr;132(4):1420-31. Epub 2007 Jan 25.


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