Literature DB >> 15128891

Advances in the treatment of mucopolysaccharidosis type I.

Joseph Muenzer1, Amy Fisher.   

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Year:  2004        PMID： 15128891     DOI： 10.1056/NEJMp048084

Source DB:  PubMed          Journal:  N Engl J Med        ISSN： 0028-4793            Impact factor:   91.245


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  16 in total

1.  Heparan sulfate levels in mucopolysaccharidoses and mucolipidoses.

Authors:  S Tomatsu; M A Gutierrez; T Ishimaru; O M Peña; A M Montaño; H Maeda; S Velez-Castrillon; T Nishioka; A A Fachel; A Cooper; M Thornley; E Wraith; L A Barrera; L S Laybauer; R Giugliani; I V Schwartz; G Schulze Frenking; M Beck; S G Kircher; E Paschke; S Yamaguchi; K Ullrich; K Isogai; Y Suzuki; T Orii; A Noguchi
Journal:  J Inherit Metab Dis       Date:  2005       Impact factor: 4.982

2.  Expression in CHO cells and pharmacokinetics and brain uptake in the Rhesus monkey of an IgG-iduronate-2-sulfatase fusion protein.

Authors:  Jeff Zhiqiang Lu; Ruben J Boado; Eric K-W Hui; Qing-Hui Zhou; William M Pardridge
Journal:  Biotechnol Bioeng       Date:  2011-03-15       Impact factor: 4.530

3.  Prevalence and development of orthopaedic symptoms in the dutch hurler patient population after haematopoietic stem cell transplantation.

Authors:  F J Stoop; M C Kruyt; M H van der Linden; R J B Sakkers; P M van Hasselt; R M C Castelein
Journal:  JIMD Rep       Date:  2012-09-19

Review 4.  Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.

Authors:  R Kohan; I A Cismondi; A M Oller-Ramirez; N Guelbert; Tapia V Anzolini; G Alonso; S E Mole; Dodelson R de Kremer; Noher I de Halac
Journal:  Curr Pharm Biotechnol       Date:  2011-06       Impact factor: 2.837

5.  AGT-181: expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys.

Authors:  Ruben J Boado; Eric K-W Hui; Jeff Zhiqiang Lu; William M Pardridge
Journal:  J Biotechnol       Date:  2009-09-06       Impact factor: 3.307

6.  Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

Authors:  Ilaria Visigalli; Stefania Delai; Letterio S Politi; Carmela Di Domenico; Federica Cerri; Emanuela Mrak; Raffaele D'Isa; Daniela Ungaro; Merel Stok; Francesca Sanvito; Elisabetta Mariani; Lidia Staszewsky; Claudia Godi; Ilaria Russo; Francesca Cecere; Ubaldo Del Carro; Alessandro Rubinacci; Riccardo Brambilla; Angelo Quattrini; Paola Di Natale; Katherine Ponder; Luigi Naldini; Alessandra Biffi
Journal:  Blood       Date:  2010-09-16       Impact factor: 22.113

7.  α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine trafficking.

Authors:  Michael J Passineau; Timothy Fahrenholz; Laurie Machen; Lee Zourelias; Katherine Nega; Rachel Paul; Mary J MacDougall; Olga Mamaeva; Richard Steet; Jarrod Barnes; H M Kingston; Raymond L Benza
Journal:  Hum Gene Ther       Date:  2011-01-27       Impact factor: 5.695

8.  Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery.

Authors:  Haiyan Fu; Julianne Dirosario; Smruti Killedar; Kimberly Zaraspe; Douglas M McCarty
Journal:  Mol Ther       Date:  2011-03-08       Impact factor: 11.454

9.  Alder-Reilly Anomaly in Hurler's Syndrome in a Neonate: A Rare Case Report.

Authors:  Pallavi Bhuyan; Bipsa Singh; Sukumar Chakrabarty; Niranjan Mohanty; Mukesh Agarwal; Sanghamitra Satpathy
Journal:  Indian J Hematol Blood Transfus       Date:  2012-07-17       Impact factor: 0.900

10.  An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT).

Authors:  Elsa Shapiro; O Evren Guler; Kyle Rudser; Kathleen Delaney; Kendra Bjoraker; Chester Whitley; Jakub Tolar; Paul Orchard; James Provenzale; Kathleen M Thomas
Journal:  Mol Genet Metab       Date:  2012-07-20       Impact factor: 4.797
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