Literature DB >> 3083887

Bone marrow transplantation for Hurler syndrome: assessment of metabolic correction.

C B Whitley, N K Ramsay, J H Kersey, W Krivit.   

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Year:  1986        PMID： 3083887

Source DB:  PubMed          Journal:  Birth Defects Orig Artic Ser        ISSN： 0547-6844


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  19 in total

1.  Marrow stromal cells from patients affected by MPS I differentially support haematopoietic progenitor cell development.

Authors:  M A Baxter; R F Wynn; L Schyma; D K Holmes; J E Wraith; L J Fairbairn; I Bellantuono
Journal:  J Inherit Metab Dis       Date:  2005       Impact factor: 4.982

Review 2.  Gene therapy for Fabry disease.

Authors:  C Siatskas; J A Medin
Journal:  J Inherit Metab Dis       Date:  2001       Impact factor: 4.982

3.  Reliability of a visual test for the rapid detection of mucopolysaccharidoses: GAG-test(®).

Authors:  Sergio Lage; José A Prieto; Fernando Andrade; Amaia Sojo; Pablo Sanjurjo; Luis J Aldámiz-Echevarría
Journal:  J Clin Lab Anal       Date:  2011       Impact factor: 2.352

4.  Standardization of α-L-iduronidase enzyme assay with Michaelis-Menten kinetics.

Authors:  Li Ou; Tyler L Herzog; Carrie M Wilmot; Chester B Whitley
Journal:  Mol Genet Metab       Date:  2013-11-26       Impact factor: 4.797

5.  A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.

Authors:  Li Ou; Michael J Przybilla; Ozan Ahlat; Sarah Kim; Paula Overn; Jeanine Jarnes; M Gerard O'Sullivan; Chester B Whitley
Journal:  Mol Ther       Date:  2020-04-08       Impact factor: 11.454

6.  Enzyme replacement in a canine model of Hurler syndrome.

Authors:  R M Shull; E D Kakkis; M F McEntee; S A Kania; A J Jonas; E F Neufeld
Journal:  Proc Natl Acad Sci U S A       Date:  1994-12-20       Impact factor: 11.205

7.  Bone marrow transplant in a case of mucopolysaccharidosis I Scheie phenotype: skin ultrastructure before and after transplantation.

Authors:  C Navarro; C Dominguez; M Costa; J J Ortega
Journal:  Acta Neuropathol       Date:  1991       Impact factor: 17.088

8.  Hematopoietic cell transplantation in murine globoid cell leukodystrophy (the twitcher mouse): effects on levels of galactosylceramidase, psychosine, and galactocerebrosides.

Authors:  T Ichioka; Y Kishimoto; S Brennan; G W Santos; A M Yeager
Journal:  Proc Natl Acad Sci U S A       Date:  1987-06       Impact factor: 11.205

9.  Long-term clinical progress in bone marrow transplanted mucopolysaccharidosis type I patients with a defined genotype.

Authors:  J J Hopwood; A Vellodi; H S Scott; C P Morris; T Litjens; P R Clements; D A Brooks; A Cooper; J E Wraith
Journal:  J Inherit Metab Dis       Date:  1993       Impact factor: 4.982

10.  Effect of bone marrow transplantation on enzyme levels and clinical course in the neurologically affected twitcher mouse.

Authors:  P M Hoogerbrugge; B J Poorthuis; A E Romme; J J van de Kamp; G Wagemaker; D W van Bekkum
Journal:  J Clin Invest       Date:  1988-06       Impact factor: 14.808
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