235 related articles for article (PubMed ID: 19384290)
1. Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system.
Aronovich EL; Bell JB; Khan SA; Belur LR; Gunther R; Koniar B; Schachern PA; Parker JB; Carlson CS; Whitley CB; McIvor RS; Gupta P; Hackett PB
Mol Ther; 2009 Jul; 17(7):1136-44. PubMed ID: 19384290
[TBL] [Abstract][Full Text] [Related]
2. Quantitative analysis of α-L-iduronidase expression in immunocompetent mice treated with the Sleeping Beauty transposon system.
Aronovich EL; Hall BC; Bell JB; McIvor RS; Hackett PB
PLoS One; 2013; 8(10):e78161. PubMed ID: 24205141
[TBL] [Abstract][Full Text] [Related]
3. Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.
Aronovich EL; Bell JB; Belur LR; Gunther R; Koniar B; Erickson DC; Schachern PA; Matise I; McIvor RS; Whitley CB; Hackett PB
J Gene Med; 2007 May; 9(5):403-15. PubMed ID: 17407189
[TBL] [Abstract][Full Text] [Related]
4. Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I.
Osborn MJ; McElmurry RT; Lees CJ; DeFeo AP; Chen ZY; Kay MA; Naldini L; Freeman G; Tolar J; Blazar BR
Mol Ther; 2011 Mar; 19(3):450-60. PubMed ID: 21081900
[TBL] [Abstract][Full Text] [Related]
5. Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.
Herati RS; Ma X; Tittiger M; Ohlemiller KK; Kovacs A; Ponder KP
J Gene Med; 2008 Sep; 10(9):972-82. PubMed ID: 18613275
[TBL] [Abstract][Full Text] [Related]
6. 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. PubMed ID: 15194053
[TBL] [Abstract][Full Text] [Related]
7. Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice.
Chung S; Ma X; Liu Y; Lee D; Tittiger M; Ponder KP
Mol Genet Metab; 2007 Feb; 90(2):181-92. PubMed ID: 16979922
[TBL] [Abstract][Full Text] [Related]
8. Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy.
Di Domenico C; Di Napoli D; Gonzalez Y Reyero E; Lombardo A; Naldini L; Di Natale P
Hum Gene Ther; 2006 Nov; 17(11):1112-21. PubMed ID: 17044753
[TBL] [Abstract][Full Text] [Related]
9. 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. PubMed ID: 15703491
[TBL] [Abstract][Full Text] [Related]
10. Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product.
Osborn MJ; McElmurry RT; Peacock B; Tolar J; Blazar BR
Mol Ther; 2008 Aug; 16(8):1459-66. PubMed ID: 18523448
[TBL] [Abstract][Full Text] [Related]
11. Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice.
Schuh RS; Gonzalez EA; Tavares AMV; Seolin BG; Elias LS; Vera LNP; Kubaski F; Poletto E; Giugliani R; Teixeira HF; Matte U; Baldo G
Gene Ther; 2020 Feb; 27(1-2):74-84. PubMed ID: 31827259
[TBL] [Abstract][Full Text] [Related]
12. Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.
Belur LR; Temme A; Podetz-Pedersen KM; Riedl M; Vulchanova L; Robinson N; Hanson LR; Kozarsky KF; Orchard PJ; Frey WH; Low WC; McIvor RS
Hum Gene Ther; 2017 Jul; 28(7):576-587. PubMed ID: 28462595
[TBL] [Abstract][Full Text] [Related]
13. Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease.
Aronovich EL; Hyland KA; Hall BC; Bell JB; Olson ER; Rusten MU; Hunter DW; Ellinwood NM; McIvor RS; Hackett PB
Hum Gene Ther; 2017 Jul; 28(7):551-564. PubMed ID: 28530135
[TBL] [Abstract][Full Text] [Related]
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. PubMed ID: 15851016
[TBL] [Abstract][Full Text] [Related]
15. Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow.
Zheng Y; Rozengurt N; Ryazantsev S; Kohn DB; Satake N; Neufeld EF
Mol Genet Metab; 2003 Aug; 79(4):233-44. PubMed ID: 12948739
[TBL] [Abstract][Full Text] [Related]
16. Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.
Ma X; Liu Y; Tittiger M; Hennig A; Kovacs A; Popelka S; Wang B; Herati R; Bigg M; Ponder KP
Mol Ther; 2007 May; 15(5):889-902. PubMed ID: 17311010
[TBL] [Abstract][Full Text] [Related]
17. Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.
Baldo G; Wozniak DF; Ohlemiller KK; Zhang Y; Giugliani R; Ponder KP
J Inherit Metab Dis; 2013 May; 36(3):499-512. PubMed ID: 22983812
[TBL] [Abstract][Full Text] [Related]
18. Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation.
Wang D; Shukla C; Liu X; Schoeb TR; Clarke LA; Bedwell DM; Keeling KM
Mol Genet Metab; 2010 Jan; 99(1):62-71. PubMed ID: 19751987
[TBL] [Abstract][Full Text] [Related]
19. Long-term nonsense suppression therapy moderates MPS I-H disease progression.
Gunn G; Dai Y; Du M; Belakhov V; Kandasamy J; Schoeb TR; Baasov T; Bedwell DM; Keeling KM
Mol Genet Metab; 2014 Mar; 111(3):374-381. PubMed ID: 24411223
[TBL] [Abstract][Full Text] [Related]
20. Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.
Wang D; Zhang W; Kalfa TA; Grabowski G; Davies S; Malik P; Pan D
Proc Natl Acad Sci U S A; 2009 Nov; 106(47):19958-63. PubMed ID: 19903883
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]