Below are the most recent publications written about "Iduronidase" by people in Profiles.
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Grady LO, Zoltick ES, Zouk H, He W, Perez E, Clarke L, Gold J, Strong A, Sahai I, Yeo J, Green RC, Karaa A, Gold NB. Long-Term Health Outcomes of Individuals With Pseudodeficiency Alleles in IDUA May Inform Newborn Screening Practices for Mucopolysaccharidosis Type I. Am J Med Genet A. 2025 Apr; 197(4):e63940.
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Wang D, Xue X, Gunn G, Du M, Siddiqui A, Weetall M, Keeling KM. Ataluren suppresses a premature termination codon in an MPS I-H mouse. J Mol Med (Berl). 2022 08; 100(8):1223-1235.
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Wang D, Belakhov V, Kandasamy J, Baasov T, Li SC, Li YT, Bedwell DM, Keeling KM. The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse. Mol Genet Metab. 2012 Jan; 105(1):116-25.
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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. Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood. 2010 Dec 09; 116(24):5130-9.
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Wang D, Shukla C, Liu X, Schoeb TR, Clarke LA, Bedwell DM, Keeling KM. Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation. Mol Genet Metab. 2010 Jan; 99(1):62-71.
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Ciron C, Cressant A, Roux F, Raoul S, Cherel Y, Hantraye P, D?glon N, Schwartz B, Barkats M, Heard JM, Tardieu M, Moullier P, Colle MA. Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution. Hum Gene Ther. 2009 Apr; 20(4):350-60.
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Ciron C, Desmaris N, Colle MA, Raoul S, Joussemet B, V?rot L, Ausseil J, Froissart R, Roux F, Ch?rel Y, Ferry N, Lajat Y, Schwartz B, Vanier MT, Maire I, Tardieu M, Moullier P, Heard JM. Gene therapy of the brain in the dog model of Hurler's syndrome. Ann Neurol. 2006 Aug; 60(2):204-13.
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Moullier P, Salvetti A, Bohl D, Danos O, Heard JM. [Gene therapy in lysosomal diseases]. C R Seances Soc Biol Fil. 1996; 190(1):45-51.
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Salvetti A, Moullier P, Cornet V, Brooks D, Hopwood JJ, Danos O, Heard JM. In vivo delivery of human alpha-L-iduronidase in mice implanted with neo-organs. Hum Gene Ther. 1995 Sep; 6(9):1153-9.