Literature DB >> 14516901

Usefulness of bone marrow transplantation in the Hurler syndrome.

Elizabeth A Braunlin1, Nanci R Stauffer, Charles H Peters, John L Bass, James M Berry, John J Hopwood, William Krivit.   

Abstract

The Hurler syndrome, an autosomal recessive storage disease of childhood, leads to death within the first decade of life from progressive deposition of glycosaminoglycans within the myointima of the coronary arteries and airways. Cardiac ultrasound findings of patients with this syndrome >10 years after successful bone marrow transplantation are described.

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Mesh：

Year:  2003        PMID： 14516901     DOI： 10.1016/s0002-9149(03)00909-3

Source DB:  PubMed          Journal:  Am J Cardiol        ISSN： 0002-9149            Impact factor:   2.778


  32 in total

1.  Pathogenesis of aortic dilatation in mucopolysaccharidosis VII mice may involve complement activation.

Authors:  Guilherme Baldo; Susan Wu; Ruth A Howe; Meera Ramamoothy; Russell H Knutsen; Jiali Fang; Robert P Mecham; Yuli Liu; Xiaobo Wu; John P Atkinson; Katherine P Ponder
Journal:  Mol Genet Metab       Date:  2011-08-24       Impact factor: 4.797

2.  Rheumatologic aspects of lysosomal storage diseases.

Authors:  Bernhard Manger; Eugen Mengel; Roland M Schaefer
Journal:  Clin Rheumatol       Date:  2006-05-06       Impact factor: 2.980

3.  Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.

Authors:  Christian Hinderer; Peter Bell; Brittney L Gurda; Qiang Wang; Jean-Pierre Louboutin; Yanqing Zhu; Jessica Bagel; Patricia O'Donnell; Tracey Sikora; Therese Ruane; Ping Wang; Mark E Haskins; James M Wilson
Journal:  Proc Natl Acad Sci U S A       Date:  2014-09-29       Impact factor: 11.205

4.  Cardiac Ultrasound Findings in Infants with Severe (Hurler Phenotype) Untreated Mucopolysaccharidosis (MPS) Type I.

Authors:  L Schroeder; P Orchard; C B Whitley; J M Berry; J Tolar; W Miller; E A Braunlin
Journal:  JIMD Rep       Date:  2013-02-12

5.  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 6.  Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.

Authors:  Madeleine Taylor; Shaukat Khan; Molly Stapleton; Jianmin Wang; Jing Chen; Robert Wynn; Hiromasa Yabe; Yasutsugu Chinen; Jaap Jan Boelens; Robert W Mason; Francyne Kubaski; Dafne D G Horovitz; Anneliese L Barth; Marta Serafini; Maria Ester Bernardo; Hironori Kobayashi; Kenji E Orii; Yasuyuki Suzuki; Tadao Orii; Shunji Tomatsu
Journal:  Biol Blood Marrow Transplant       Date:  2019-02-14       Impact factor: 5.742

7.  Fatal and unanticipated cardiorespiratory disease in a two-year-old child with hurler syndrome following successful stem cell transplant.

Authors:  Sampada Gupta; Anne O'Meara; Robert Wynn; Michael McDermott
Journal:  JIMD Rep       Date:  2013-03-09

Review 8.  Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases.

Authors:  William Krivit
Journal:  Springer Semin Immunopathol       Date:  2004-09-25

9.  Gender-related dimorphism in aortic insufficiency in murine mucopolysaccharidosis type I.

Authors:  Jakub Tolar; Elizabeth Braunlin; Megan Riddle; Brandon Peacock; Ron T McElmurry; Paul J Orchard; Bruce R Blazar
Journal:  J Heart Valve Dis       Date:  2009-09

10.  alpha-L-iduronidase therapy for mucopolysaccharidosis type I.

Authors:  Jakub Tolar; Paul J Orchard
Journal:  Biologics       Date:  2008-12
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