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

Author:

Hopwood J. J.,Vellodi A.,Scott H. S.,Morris C. P.,Litjens T.,Clements P. R.,Brooks D. A.,Cooper A.,Wraith J. E.

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Reference15 articles.

1. Ashton LJ, Brooks DA, McCourt PAG, Muller VJ, Clements PR, Hopwood JJ (1992) Immunoquantification and enzyme kinetics of ?-L-iduronidase in cultured fibroblasts for mucopolysaccharidosis type I patients.Am J Hum Genet 50: 789?794.

2. Hobbs JR, Barrett AJ, Chambers D et al (1981) Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation.Lancet 2: 709?712.

3. Hoogerbrugge PM, Vossen JMJJ (1990) In Fernandes J, Saudubray J-M, Tada K, eds.Inborn Metabolic Diseases. New York, Springer-Verlag, 659?670.

4. Hopwood JJ, Harrison JR (1982) High resolution electrophoresis of urinary glycosaminoglycans.Anal Biochem 199: 120?127.

5. Hopwood JJ, Morris CP (1990) The mucopolysaccharidoses: diagnosis, molecular genetics and treatment.Mol Biol Med 7: 381?404.

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