306 related articles for article (PubMed ID: 9425437)
1. Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts.
Huang MM; Wong A; Yu X; Kakkis E; Kohn DB
Gene Ther; 1997 Nov; 4(11):1150-9. PubMed ID: 9425437
[TBL] [Abstract][Full Text] [Related]
2. Retroviral vector design studies toward hematopoietic stem cell gene therapy for mucopolysaccharidosis type I.
Pan D; Aronovich E; McIvor RS; Whitley CB
Gene Ther; 2000 Nov; 7(21):1875-83. PubMed ID: 11110421
[TBL] [Abstract][Full Text] [Related]
3. 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]
4. Towards gene therapy of Hurler syndrome.
Fairbairn LJ; Lashford LS; Spooncer E; McDermott RH; Lebens G; Arrand JE; Arrand JR; Bellantuono I; Holt R; Hatton CE; Cooper A; Besley GT; Wraith JE; Anson DS; Hopwood JJ; Dexter TM
Cas Lek Cesk; 1997 Jan; 136(1):27-31. PubMed ID: 9127508
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. Improved gene transfer and normalized enzyme levels in primitive hematopoietic progenitors from patients with mucopolysaccharidosis type I using a bioreactor.
Pan D; Stroncek DF; Whitley CB
J Gene Med; 2004 Dec; 6(12):1293-303. PubMed ID: 15538732
[TBL] [Abstract][Full Text] [Related]
7. Mobilization and transduction of peripheral blood progenitor cells in patients with mucopolysaccharidosis I.
Hubel A; Stroncek D; Pan D; Whitley CB; McCullough J
J Hematother; 1998 Dec; 7(6):505-14. PubMed ID: 9919944
[TBL] [Abstract][Full Text] [Related]
8. 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. PubMed ID: 16482204
[TBL] [Abstract][Full Text] [Related]
9. Prevention of neuropathology in the mouse model of Hurler syndrome.
Desmaris N; Verot L; Puech JP; Caillaud C; Vanier MT; Heard JM
Ann Neurol; 2004 Jul; 56(1):68-76. PubMed ID: 15236403
[TBL] [Abstract][Full Text] [Related]
10. Identification and molecular characterization of alpha-L-iduronidase mutations present in mucopolysaccharidosis type I patients undergoing enzyme replacement therapy.
Yogalingam G; Guo XH; Muller VJ; Brooks DA; Clements PR; Kakkis ED; Hopwood JJ
Hum Mutat; 2004 Sep; 24(3):199-207. PubMed ID: 15300847
[TBL] [Abstract][Full Text] [Related]
11. Residual α-L-iduronidase activity in fibroblasts of mild to severe Mucopolysaccharidosis type I patients.
Oussoren E; Keulemans J; van Diggelen OP; Oemardien LF; Timmermans RG; van der Ploeg AT; Ruijter GJ
Mol Genet Metab; 2013 Aug; 109(4):377-81. PubMed ID: 23786846
[TBL] [Abstract][Full Text] [Related]
12. Gene therapy for canine alpha-L-iduronidase deficiency: in utero adoptive transfer of genetically corrected hematopoietic progenitors results in engraftment but not amelioration of disease.
Lutzko C; Omori F; Abrams-Ogg AC; Shull R; Li L; Lau K; Ruedy C; Nanji S; Gartley C; Dobson H; Foster R; Kruth S; Dubé ID
Hum Gene Ther; 1999 Jun; 10(9):1521-32. PubMed ID: 10395377
[TBL] [Abstract][Full Text] [Related]
13. Sustained transgene expression by human cord blood derived CD34+ cells transduced with simian immunodeficiency virus agmTYO1-based vectors carrying the human coagulation factor VIII gene in NOD/SCID mice.
Kikuchi J; Mimuro J; Ogata K; Tabata T; Ueda Y; Ishiwata A; Kimura K; Takano K; Madoiwa S; Mizukami H; Hanazono Y; Kume A; Hasegawa M; Ozawa K; Sakata Y
J Gene Med; 2004 Oct; 6(10):1049-60. PubMed ID: 15386735
[TBL] [Abstract][Full Text] [Related]
14. Retroviral-mediated gene transfer into mammalian cells.
Kohn DB; Kantoff PW; Eglitis MA; McLachlin JR; Moen RC; Karson E; Zwiebel JA; Nienhuis A; Karlsson S; O'Reilly R
Blood Cells; 1987; 13(1-2):285-98. PubMed ID: 3311223
[TBL] [Abstract][Full Text] [Related]
15. Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells.
Kakkis ED; Matynia A; Jonas AJ; Neufeld EF
Protein Expr Purif; 1994 Jun; 5(3):225-32. PubMed ID: 7950365
[TBL] [Abstract][Full Text] [Related]
16. Congenital erythropoietic porphyria: prolonged high-level expression and correction of the heme biosynthetic defect by retroviral-mediated gene transfer into porphyric and erythroid cells.
Kauppinen R; Glass IA; Aizencang G; Astrin KH; Atweh GF; Desnick RJ
Mol Genet Metab; 1998 Sep; 65(1):10-7. PubMed ID: 9787090
[TBL] [Abstract][Full Text] [Related]
17. Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.
Fairbairn LJ; Lashford LS; Spooncer E; McDermott RH; Lebens G; Arrand JE; Arrand JR; Bellantuono I; Holt R; Hatton CE; Cooper A; Besley GT; Wraith JE; Anson DS; Hopwood JJ; Dexter TM
Proc Natl Acad Sci U S A; 1996 Mar; 93(5):2025-30. PubMed ID: 8700879
[TBL] [Abstract][Full Text] [Related]
18. Uptake of alpha-(L)-iduronidase produced by retrovirally transduced fibroblasts into neuronal and glial cells in vitro.
Stewart K; Brown OA; Morelli AE; Fairbairn LJ; Lashford LS; Cooper A; Hatton CE; Dexter TM; Castro MG; Lowenstein PR
Gene Ther; 1997 Jan; 4(1):63-75. PubMed ID: 9068797
[TBL] [Abstract][Full Text] [Related]
19. Correction of mucopolysaccharidosis type I fibroblasts by retroviral-mediated transfer of the human alpha-L-iduronidase gene.
Anson DS; Bielicki J; Hopwood JJ
Hum Gene Ther; 1992 Aug; 3(4):371-9. PubMed ID: 1525210
[TBL] [Abstract][Full Text] [Related]
20. Construction of a high efficiency retroviral vector for gene therapy of Hunter's syndrome.
Hong Y; Yu SS; Kim JM; Lee K; Na YS; Whitley CB; Sugimoto Y; Kim S
J Gene Med; 2003 Jan; 5(1):18-29. PubMed ID: 12516048
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]