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Murine MPS I: insights into the pathogenesis of Hurler syndrome.

Russell C, Hendson G, Jevon G, Matlock T, Yu J, Aklujkar M, Ng KY, Clarke LA.

Clin Genet. 1998 May;53(5):349-61.


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. doi: 10.1016/j.ymgme.2013.09.008. Epub 2013 Sep 19.


Lipid composition of whole brain and cerebellum in Hurler syndrome (MPS IH) mice.

Heinecke KA, Peacock BN, Blazar BR, Tolar J, Seyfried TN.

Neurochem Res. 2011 Sep;36(9):1669-76. doi: 10.1007/s11064-011-0400-y. Epub 2011 Jan 21.


Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I.

Pievani A, Azario I, Antolini L, Shimada T, Patel P, Remoli C, Rambaldi B, Valsecchi MG, Riminucci M, Biondi A, Tomatsu S, Serafini M.

Blood. 2015 Mar 5;125(10):1662-71. doi: 10.1182/blood-2014-06-581207. Epub 2014 Oct 8.


Alterations of membrane lipids and in gene expression of ganglioside metabolism in different brain structures in a mouse model of mucopolysaccharidosis type I (MPS I).

Kreutz F, dos Santos Petry F, Camassola M, Schein V, Guma FC, Nardi NB, Trindade VM.

Gene. 2013 Sep 15;527(1):109-14. doi: 10.1016/j.gene.2013.06.002. Epub 2013 Jun 15.


Early onset of lysosomal storage disease in a murine model of mucopolysaccharidosis type VII: undegraded substrate accumulates in many tissues in the fetus and very young MPS VII mouse.

Vogler C, Levy B, Galvin N, Lessard M, Soper B, Barker J.

Pediatr Dev Pathol. 2005 Jul-Aug;8(4):453-62. Epub 2005 Oct 12.


Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations.

Vite CH, Wang P, Patel RT, Walton RM, Walkley SU, Sellers RS, Ellinwood NM, Cheng AS, White JT, O'Neill CA, Haskins M.

Mol Genet Metab. 2011 Jul;103(3):268-74. doi: 10.1016/j.ymgme.2011.03.011. Epub 2011 Mar 21.


Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene.

Clarke LA, Russell CS, Pownall S, Warrington CL, Borowski A, Dimmick JE, Toone J, Jirik FR.

Hum Mol Genet. 1997 Apr;6(4):503-11.


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. doi: 10.1227/NEU.0000000000000157.


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):2028-37. doi: 10.1038/mt.2014.152. Epub 2014 Aug 4.


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 Sep-Oct;83(1-2):163-74.


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 9;116(24):5130-9. doi: 10.1182/blood-2010-04-278234. Epub 2010 Sep 16.


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 24;106(47):19958-63. doi: 10.1073/pnas.0908528106. Epub 2009 Nov 10.


Mucopolysaccharidosis type I.

Wraith JE, Jones S.

Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:102-6. Review.


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.


Evidence of a progressive motor dysfunction in Mucopolysaccharidosis type I mice.

Baldo G, Mayer FQ, Martinelli B, Dilda A, Meyer F, Ponder KP, Giugliani R, Matte U.

Behav Brain Res. 2012 Jul 15;233(1):169-75. doi: 10.1016/j.bbr.2012.04.051. Epub 2012 May 9.


Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy.

Garcia-Rivera MF, Colvin-Wanshura LE, Nelson MS, Nan Z, Khan SA, Rogers TB, Maitra I, Low WC, Gupta P.

Brain Res Bull. 2007 Nov 1;74(6):429-38. Epub 2007 Aug 6. Erratum in: Brain Res Bull. 2008 Aug 15;76(6):640-1.


Clinical features of Mexican patients with Mucopolysaccharidosis type I.

Alonzo-Rojo A, García-Ortiz JE, Ortiz-Aranda M, Gallegos-Arreola MP, Figuera-Villanueva LE.

Genet Mol Res. 2017 Sep 21;16(3). doi: 10.4238/gmr16032602.


Cardiac functional and histopathologic findings in humans and mice with mucopolysaccharidosis type I: implications for assessment of therapeutic interventions in hurler syndrome.

Braunlin E, Mackey-Bojack S, Panoskaltsis-Mortari A, Berry JM, McElmurry RT, Riddle M, Sun LY, Clarke LA, Tolar J, Blazar BR.

Pediatr Res. 2006 Jan;59(1):27-32. Epub 2005 Dec 2.


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