These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

251 related articles for article (PubMed ID: 21397026)

  • 21. Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.
    Dierenfeld AD; McEntee MF; Vogler CA; Vite CH; Chen AH; Passage M; Le S; Shah S; Jens JK; Snella EM; Kline KL; Parkes JD; Ware WA; Moran LE; Fales-Williams AJ; Wengert JA; Whitley RD; Betts DM; Boal AM; Riedesel EA; Gross W; Ellinwood NM; Dickson PI
    Sci Transl Med; 2010 Dec; 2(60):60ra89. PubMed ID: 21123810
    [TBL] [Abstract][Full Text] [Related]  

  • 22. Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice.
    Hinderer C; Katz N; Louboutin JP; Bell P; Yu H; Nayal M; Kozarsky K; O'Brien WT; Goode T; Wilson JM
    Hum Gene Ther; 2016 Nov; 27(11):906-915. PubMed ID: 27510804
    [TBL] [Abstract][Full Text] [Related]  

  • 23. Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I.
    Belur LR; Romero M; Lee J; Podetz-Pedersen KM; Nan Z; Riedl MS; Vulchanova L; Kitto KF; Fairbanks CA; Kozarsky KF; Orchard PJ; Frey WH; Low WC; McIvor RS
    Front Mol Neurosci; 2021; 14():618360. PubMed ID: 34040503
    [TBL] [Abstract][Full Text] [Related]  

  • 24. 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]  

  • 25. Mucopolysaccharidoses type I gene therapy.
    Hurt SC; Dickson PI; Curiel DT
    J Inherit Metab Dis; 2021 Sep; 44(5):1088-1098. PubMed ID: 34189746
    [TBL] [Abstract][Full Text] [Related]  

  • 26. In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression.
    Meertens L; Zhao Y; Rosic-Kablar S; Li L; Chan K; Dobson H; Gartley C; Lutzko C; Hopwood J; Kohn D; Kruth S; Hough MR; Dubé ID
    Hum Gene Ther; 2002 Oct; 13(15):1809-20. PubMed ID: 12396614
    [TBL] [Abstract][Full Text] [Related]  

  • 27. A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.
    Ou L; Przybilla MJ; Ahlat O; Kim S; Overn P; Jarnes J; O'Sullivan MG; Whitley CB
    Mol Ther; 2020 Jun; 28(6):1442-1454. PubMed ID: 32278382
    [TBL] [Abstract][Full Text] [Related]  

  • 28. 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]  

  • 29. Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model.
    Hinderer C; Bell P; Louboutin JP; Katz N; Zhu Y; Lin G; Choa R; Bagel J; O'Donnell P; Fitzgerald CA; Langan T; Wang P; Casal ML; Haskins ME; Wilson JM
    Mol Genet Metab; 2016 Sep; 119(1-2):124-30. PubMed ID: 27386755
    [TBL] [Abstract][Full Text] [Related]  

  • 30. Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies.
    Visigalli I; Delai S; Ferro F; Cecere F; Vezzoli M; Sanvito F; Chanut F; Benedicenti F; Spinozzi G; Wynn R; Calabria A; Naldini L; Montini E; Cristofori P; Biffi A
    Hum Gene Ther; 2016 Oct; 27(10):813-829. PubMed ID: 27431943
    [TBL] [Abstract][Full Text] [Related]  

  • 31. Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.
    Dickson P; Peinovich M; McEntee M; Lester T; Le S; Krieger A; Manuel H; Jabagat C; Passage M; Kakkis ED
    J Clin Invest; 2008 Aug; 118(8):2868-76. PubMed ID: 18654665
    [TBL] [Abstract][Full Text] [Related]  

  • 32. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.
    Tolar J; Grewal SS; Bjoraker KJ; Whitley CB; Shapiro EG; Charnas L; Orchard PJ
    Bone Marrow Transplant; 2008 Mar; 41(6):531-5. PubMed ID: 18037941
    [TBL] [Abstract][Full Text] [Related]  

  • 33. Mucopolysaccharidosis type I.
    Wraith JE; Jones S
    Pediatr Endocrinol Rev; 2014 Sep; 12 Suppl 1():102-6. PubMed ID: 25345091
    [TBL] [Abstract][Full Text] [Related]  

  • 34. 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]  

  • 35. Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration.
    Wolf DA; Hanson LR; Aronovich EL; Nan Z; Low WC; Frey WH; McIvor RS
    Mol Genet Metab; 2012 May; 106(1):131-4. PubMed ID: 22420937
    [TBL] [Abstract][Full Text] [Related]  

  • 36. Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis 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
    Mol Ther; 2014 Dec; 22(12):2018-2027. PubMed ID: 25027660
    [TBL] [Abstract][Full Text] [Related]  

  • 37. 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. PubMed ID: 15585404
    [TBL] [Abstract][Full Text] [Related]  

  • 38. 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]  

  • 39. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I.
    Kakkis E; McEntee M; Vogler C; Le S; Levy B; Belichenko P; Mobley W; Dickson P; Hanson S; Passage M
    Mol Genet Metab; 2004; 83(1-2):163-74. PubMed ID: 15464431
    [TBL] [Abstract][Full Text] [Related]  

  • 40. 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]  

    [Previous]   [Next]    [New Search]
    of 13.