In vitro correction of iduronate-2-sulfatase deficiency by adenovirus-mediated gene transfer (original) (raw)

Decreasing Activity and Altered Protein Processing of Human Iduronate-2-sulfatase Mutations Demonstrated by Expression in COS7 Cells

Voraratt Champattanachai

Biochemical Genetics, 2012

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Intermediate form of mucopolysaccharidosis type II (Hunter disease): A C1327 to T substitution in the iduronate sulfatase gene

Katsuyuki Tamai

Biochemical and Biophysical Research Communications, 1992

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Hunter's syndrome: A deficiency of L-idurono-sulfate sulfatase

L.-a. Fransson, Reuben Matalon

Biochemical and Biophysical Research Communications, 1973

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Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase

Chester Whitley

Proceedings of the National Academy of Sciences, 1993

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Gene therapy of Hunter syndrome: Evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS)

Maurizio Scarpa

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 2008

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Identification of 6 new mutations in the iduronate sulfatase gene

Marion Coulter-mackie

Human Mutation, 1999

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Profile of idursulfase for the treatment of Hunter syndrome

Ferdinando Ceravolo

Research and Reports in Endocrine Disorders, 2015

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Uptake of recombinant iduronate-2-sulfatase into neuronal and glial cells in vitro

Aurora Daniele

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 2002

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Bioinformatic Analysis of the Human Recombinant Iduronate 2-Sulfate Sulfatase

Patricia Landazuri

The Open Microbiology Journal, 2016

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The effect of four mutations on the expression of iduronate-2-sulfatase in mucopolysaccharidosis type II

Mirella Filocamo, Stefano Regis

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Expression of five iduronate-2-sulfatase site-directed mutations

Nicola Balzano

Biochimica Et Biophysica Acta-molecular Basis of Disease, 2000

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Murine model (Galnstm(C76S)slu) of MPS IVA with missense mutation at the active site cysteine conserved among sulfatase proteins

MONICA GUTIERREZ

Molecular Genetics and Metabolism, 2007

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The correction of hunter fibroblasts by exogenous iduronate sulfate sulfatase: Biochemical and ultrastructural studies

Gideon Bach

The American Journal of Human Genetics, 1981

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Hunter syndrome: isolation of an iduronate-2-sulfatase cDNA clone and analysis of patient DNA

peter Clements

Proceedings of the National Academy of Sciences, 1990

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The Open Microbiology Journal Bioinformatic Analysis of the Human Recombinant Iduronate 2- Sulfate Sulfatase

Raúl Alberto Poutou Piñales

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The Effect of Recombinant Human Iduronate-2-Sulfatase (Idursulfase) on Growth in Young Patients with Mucopolysaccharidosis Type II

Anna Tylki-szymanska

PLoS ONE, 2014

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Mutational spectrum of the iduronate 2 sulfatase gene in 25 unrelated Korean Hunter syndrome patients: Identification of 13 novel mutations

Cheol Kyu Han

Human Mutation, 2003

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Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)

Regis Andriolo

… Database Syst Rev, 2011

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Molecular analysis of the iduronate-2-sulfatase gene in Thai patients with Hunter syndrome

James Ketudat Cairns

Journal of Inherited Metabolic Disease, 2008

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Successful desensitization to idursulfase in a patient with type II mucopolysaccharidosis (Hunter syndrome)

Carlos Serrano

Journal of investigational allergology & clinical immunology, 2011

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Molecular basis of mucopolysaccharidosis type II: Mutations in the iduronate-2-sulphatase gene

Rudee Surarit

Human Mutation, 1993

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Molecular analysis of 40 Italian patients with mucopolysaccharidosis type II: New mutations in the iduronate-2-sulfatase (IDS) gene

Mirella Filocamo

Human Mutation, 2001

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Comparative study of idursulfase beta and idursulfase in vitro and in vivo

Chihwa Kim

Journal of Human Genetics, 2016

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Enigmatic In Vivo iduronate-2-sulfatase (IDS) mutant transcript correction to wild-type in Hunter syndrome

Mirella Filocamo, Susanna Lualdi, Stefano Regis

Human Mutation, 2010

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Identification of novel variation in introns three and four of the iduronate-2-sulfatase gene in patients with mucopolysaccharidosis type II

steven arianto

Journal of Physics: Conference Series, 2018

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Homologous nonallelic recombinations between the iduronate-sulfatase gene and pseudogene cause various intragenic deletions and inversions in patients with mucopolysaccharidosis type II

Ewa Popowska

European Journal of Human Genetics, 1998

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Results of enzyme replacement therapy in Bulgarian patients with a severe form of hunter syndrome: A 42-month follow-up

Radka Tincheva

Genetika, 2018

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Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy

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