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Items: 1 to 20 of 45

1.

Limitations of enzyme replacement therapy: current and future.

Wraith JE.

J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):442-7.

PMID:
16763916
2.

Structure-based prediction of insertion-site preferences of transposons into chromosomes.

Geurts AM, Hackett CS, Bell JB, Bergemann TL, Collier LS, Carlson CM, Largaespada DA, Hackett PB.

Nucleic Acids Res. 2006 May 22;34(9):2803-11. Print 2006.

3.

Cell and gene-based therapies for the lysosomal storage diseases.

Hodges BL, Cheng SH.

Curr Gene Ther. 2006 Apr;6(2):227-41. Review.

PMID:
16611044
4.

Gene therapy for lysosomal storage diseases.

Sands MS, Davidson BL.

Mol Ther. 2006 May;13(5):839-49. Epub 2006 Mar 20. Review.

5.

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice.

Watson G, Bastacky J, Belichenko P, Buddhikot M, Jungles S, Vellard M, Mobley WC, Kakkis E.

Gene Ther. 2006 Jun;13(11):917-25.

PMID:
16482204
6.
7.

Nonviral in vivo gene transfer in the mucopolysaccharidosis I murine model.

Camassola M, Braga LM, Delgado-CaƱedo A, Dalberto TP, Matte U, Burin M, Giugliani R, Nardi NB.

J Inherit Metab Dis. 2005;28(6):1035-43.

PMID:
16435197
8.

Dynamic gene expression after systemic delivery of plasmid DNA as determined by in vivo bioluminescence imaging.

Wilber A, Frandsen JL, Wangensteen KJ, Ekker SC, Wang X, McIvor RS.

Hum Gene Ther. 2005 Nov;16(11):1325-32.

PMID:
16259566
9.

Sleeping beauty transposon-mediated gene therapy for prolonged expression.

Hackett PB, Ekker SC, Largaespada DA, McIvor RS.

Adv Genet. 2005;54:189-232. Review.

PMID:
16096013
10.

Awakening gene therapy with Sleeping Beauty transposons.

Essner JJ, McIvor RS, Hackett PB.

Curr Opin Pharmacol. 2005 Oct;5(5):513-9. Review.

PMID:
16084771
11.

Mammalian mutagenesis using a highly mobile somatic Sleeping Beauty transposon system.

Dupuy AJ, Akagi K, Largaespada DA, Copeland NG, Jenkins NA.

Nature. 2005 Jul 14;436(7048):221-6.

12.

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

Franco LM, Sun B, Yang X, Bird A, Zhang H, Schneider A, Brown T, Young SP, Clay TM, Amalfitano A, Chen YT, Koeberl DD.

Mol Ther. 2005 Nov;12(5):876-84. Epub 2005 Jul 6.

13.

Cardiac manifestations in the mouse model of mucopolysaccharidosis I.

Jordan MC, Zheng Y, Ryazantsev S, Rozengurt N, Roos KP, Neufeld EF.

Mol Genet Metab. 2005 Sep-Oct;86(1-2):233-43. Epub 2005 Jun 24.

14.

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector.

Kobayashi H, Carbonaro D, Pepper K, Petersen D, Ge S, Jackson H, Shimada H, Moats R, Kohn DB.

Mol Ther. 2005 May;11(5):776-89.

15.

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

Di Domenico C, Villani GR, Di Napoli D, Reyero EG, Lombardo A, Naldini L, Di Natale P.

Hum Gene Ther. 2005 Jan;16(1):81-90.

PMID:
15703491
16.

Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice.

Liu Y, Xu L, Hennig AK, Kovacs A, Fu A, Chung S, Lee D, Wang B, Herati RS, Mosinger Ogilvie J, Cai SR, Parker Ponder K.

Mol Ther. 2005 Jan;11(1):35-47.

17.

Kupffer cells and not liver sinusoidal endothelial cells prevent lentiviral transduction of hepatocytes.

van Til NP, Markusic DM, van der Rijt R, Kunne C, Hiralall JK, Vreeling H, Frederiks WM, Oude-Elferink RP, Seppen J.

Mol Ther. 2005 Jan;11(1):26-34.

18.

Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system.

Ohlfest JR, Frandsen JL, Fritz S, Lobitz PD, Perkinson SG, Clark KJ, Nelsestuen G, Key NS, McIvor RS, Hackett PB, Largaespada DA.

Blood. 2005 Apr 1;105(7):2691-8. Epub 2004 Dec 2.

19.

Integration and long-term expression in xenografted human glioblastoma cells using a plasmid-based transposon system.

Ohlfest JR, Lobitz PD, Perkinson SG, Largaespada DA.

Mol Ther. 2004 Aug;10(2):260-8.

20.

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene.

Hartung SD, Frandsen JL, Pan D, Koniar BL, Graupman P, Gunther R, Low WC, Whitley CB, McIvor RS.

Mol Ther. 2004 Jun;9(6):866-75.

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