Literature DB >> 19324223

Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy.

Robert F Wynn1, J Ed Wraith, Jean Mercer, Anne O'Meara, Karen Tylee, Margaret Thornley, Heather J Church, Brian W Bigger.   

Abstract

We compared substrate reduction in patients with lysosomal storage disorder treated with hematopoietic stem cell transplant and found that it was significantly reduced compared with patients treated with pharmacological enzyme replacement therapy. These data might support the wider application of hematopoietic stem cell transplant in the treatment of lysosomal storage disorders.

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Year:  2009        PMID： 19324223     DOI： 10.1016/j.jpeds.2008.11.005

Source DB:  PubMed          Journal:  J Pediatr        ISSN： 0022-3476            Impact factor:   4.406


  59 in total

1.  Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.

Authors:  Muhammad Ameer Saif; Brian W Bigger; Karen E Brookes; Jean Mercer; Karen L Tylee; Heather J Church; Denise K Bonney; Simon Jones; J Ed Wraith; Robert F Wynn
Journal:  Haematologica       Date:  2012-02-27       Impact factor: 9.941

Review 2.  Research challenges in central nervous system manifestations of inborn errors of metabolism.

Authors:  P I Dickson; A R Pariser; S C Groft; R W Ishihara; D E McNeil; D Tagle; D J Griebel; S G Kaler; J W Mink; E G Shapiro; K J Bjoraker; L Krivitzky; J M Provenzale; A Gropman; P Orchard; G Raymond; B H Cohen; R D Steiner; S F Goldkind; R M Nelson; E Kakkis; M C Patterson
Journal:  Mol Genet Metab       Date:  2010-12-02       Impact factor: 4.797

3.  Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.

Authors:  Sean Turbeville; Helen Nicely; J Douglas Rizzo; Tanya L Pedersen; Paul J Orchard; Mitchell E Horwitz; Edwin M Horwitz; Paul Veys; Carmem Bonfim; Amal Al-Seraihy
Journal:  Mol Genet Metab       Date:  2010-10-25       Impact factor: 4.797

4.  Delayed hypertrophic differentiation of epiphyseal chondrocytes contributes to failed secondary ossification in mucopolysaccharidosis VII dogs.

Authors:  Sun H Peck; Philip J M O'Donnell; Jennifer L Kang; Neil R Malhotra; George R Dodge; Maurizio Pacifici; Eileen M Shore; Mark E Haskins; Lachlan J Smith
Journal:  Mol Genet Metab       Date:  2015-09-26       Impact factor: 4.797

5.  Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome).

Authors:  Jakub Tolar; In-Hyun Park; Lily Xia; Chris J Lees; Brandon Peacock; Beau Webber; Ron T McElmurry; Cindy R Eide; Paul J Orchard; Michael Kyba; Mark J Osborn; Troy C Lund; John E Wagner; George Q Daley; Bruce R Blazar
Journal:  Blood       Date:  2010-10-29       Impact factor: 22.113

Review 6.  Specific protein markers for stem cell cross-talk with neighboring cells in the environment.

Authors:  Kyung Soo Park; Seung Won Shin; Jeong-Woo Choi; Soong Ho Um
Journal:  Int J Stem Cells       Date:  2013-11       Impact factor: 2.500

7.  Barriers to transplantation in adults with inborn errors of metabolism.

Authors:  S M Sirrs; H Faghfoury; E M Yoshida; T Geberhiwot
Journal:  JIMD Rep       Date:  2012-08-22

8.  Cross correction following haemopoietic stem cell transplant for purine nucleoside phosphorylase deficiency: engrafted donor-derived white blood cells provide enzyme to residual enzyme-deficient recipient cells.

Authors:  Vikramajit Singh
Journal:  JIMD Rep       Date:  2012-03-02

9.  Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.

Authors:  Jaap Jan Boelens; Mieke Aldenhoven; Duncan Purtill; Annalisa Ruggeri; Todd Defor; Robert Wynn; Ed Wraith; Marina Cavazzana-Calvo; Attilio Rovelli; Alain Fischer; Jakub Tolar; Vinod K Prasad; Maria Escolar; Eliane Gluckman; Anne O'Meara; Paul J Orchard; Paul Veys; Mary Eapen; Joanne Kurtzberg; Vanderson Rocha
Journal:  Blood       Date:  2013-03-14       Impact factor: 22.113

Review 10.  New perspectives in human stem cell therapeutic research.

Authors:  Alan Trounson
Journal:  BMC Med       Date:  2009-06-11       Impact factor: 8.775
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