BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

256 related articles for article (PubMed ID: 21397026)

  • 1. Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.
    Wolf DA; Lenander AW; Nan Z; Belur LR; Whitley CB; Gupta P; Low WC; McIvor RS
    Neurobiol Dis; 2011 Jul; 43(1):123-33. PubMed ID: 21397026
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction.
    Podetz-Pedersen KM; Laoharawee K; Singh S; Nguyen TT; Smith MC; Temme A; Kozarsky K; McIvor RS; Belur LR
    Hum Gene Ther; 2023 Jan; 34(1-2):8-18. PubMed ID: 36541357
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease.
    Janson CG; Romanova LG; Leone P; Nan Z; Belur L; McIvor RS; Low WC
    Neurosurgery; 2014 Jan; 74(1):99-111. PubMed ID: 24077583
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
    Hinderer C; Bell P; Louboutin JP; Zhu Y; Yu H; Lin G; Choa R; Gurda BL; Bagel J; O'Donnell P; Sikora T; Ruane T; Wang P; Tarantal AF; Casal ML; Haskins ME; Wilson JM
    Mol Ther; 2015 Aug; 23(8):1298-1307. PubMed ID: 26022732
    [TBL] [Abstract][Full Text] [Related]  

  • 7. High-dose enzyme replacement therapy in murine Hurler syndrome.
    Ou L; Herzog T; Koniar BL; Gunther R; Whitley CB
    Mol Genet Metab; 2014 Feb; 111(2):116-22. PubMed ID: 24100243
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
    Visigalli I; Delai S; Politi LS; Di Domenico C; Cerri F; Mrak E; D'Isa R; Ungaro D; Stok M; Sanvito F; Mariani E; Staszewsky L; Godi C; Russo I; Cecere F; Del Carro U; Rubinacci A; Brambilla R; Quattrini A; Di Natale P; Ponder K; Naldini L; Biffi A
    Blood; 2010 Dec; 116(24):5130-9. PubMed ID: 20847202
    [TBL] [Abstract][Full Text] [Related]  

  • 9. RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system.
    Ou L; Przybilla MJ; Koniar B; Whitley CB
    Mol Genet Metab; 2018 Feb; 123(2):105-111. PubMed ID: 29198892
    [TBL] [Abstract][Full Text] [Related]  

  • 10. In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors.
    Di Natale P; Di Domenico C; Villani GR; Lombardo A; Follenzi A; Naldini L
    Eur J Biochem; 2002 Jun; 269(11):2764-71. PubMed ID: 12047386
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 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. Intracerebroventricular transplantation of human bone marrow-derived multipotent progenitor cells in an immunodeficient mouse model of mucopolysaccharidosis type I (MPS-I).
    Nan Z; Shekels L; Ryabinin O; Evavold C; Nelson MS; Khan SA; Deans RJ; Mays RW; Low WC; Gupta P
    Cell Transplant; 2012; 21(7):1577-93. PubMed ID: 22472595
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.
    El-Amouri SS; Dai M; Han JF; Brady RO; Pan D
    Mol Ther; 2014 Dec; 22(12):2028-2037. PubMed ID: 25088464
    [TBL] [Abstract][Full Text] [Related]  

  • 15. 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]  

  • 16. Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice.
    Baldo G; Mayer FQ; Martinelli BZ; de Carvalho TG; Meyer FS; de Oliveira PG; Meurer L; Tavares A; Matte U; Giugliani R
    Mol Genet Metab; 2013 May; 109(1):33-40. PubMed ID: 23562162
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.
    Hinderer C; Bell P; Gurda BL; Wang Q; Louboutin JP; Zhu Y; Bagel J; O'Donnell P; Sikora T; Ruane T; Wang P; Haskins ME; Wilson JM
    Proc Natl Acad Sci U S A; 2014 Oct; 111(41):14894-9. PubMed ID: 25267637
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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]  

  • 20. Neonatal combination therapy improves some of the clinical manifestations in the Mucopolysaccharidosis type I murine model.
    Santi L; De Ponti G; Dina G; Pievani A; Corsi A; Riminucci M; Khan S; Sawamoto K; Antolini L; Gregori S; Annoni A; Biondi A; Quattrini A; Tomatsu S; Serafini M
    Mol Genet Metab; 2020 Jul; 130(3):197-208. PubMed ID: 32439268
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.