PMID: 6410119Jan 1, 1983Paper

A clinical trial of fibroblast transplantation for the treatment of mucopolysaccharidoses

Journal of Inherited Metabolic Disease
D A GibbsJ F Mowbray

Abstract

This paper reports the clinical and biochemical results in six patients with Hurler disease (Mucopolysaccharidosis IH; McKusick 25280), two patients with Hunter disease (Mucopolysaccharidosis II; McKusick 25285) and one patient with Sanfilippo B disease (Mucopolysaccharidosis IIIB; McKusick 25292) who were treated by fibroblast transplantation. Except for one patient who died for a coincidental reason, the patients have been studied for between 2.5 and 4.5 years. The clinical course of the disease was not materially altered. There was no evidence that the patients had developed immune responses against the transplanted fibroblasts. Transplantation did not produce measurable levels of either alpha-L-iduronidase (EC 3.2.1.76) in the leukocytes from patients with Hurler disease or of N-acetyl-alpha-D-glucosaminidase (EC 3.2.1.50) in the plasma of the patients with Sanfilippo B disease. Under the conditions used for the assay, leukocytes from the patients with Hunter disease had detectable levels of residual alpha-L-idurono-2-sulphate sulphatase activity which were increased after the transplants, although these changes were of inconstant size and their time course was not consistently related to the transplantations. Cytogenetic s...Continue Reading

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Citations

Jan 1, 1986·Journal of Inherited Metabolic Disease·R W WattsJ H Adams
Jan 1, 1992·Journal of Inherited Metabolic Disease·J MuenzerJ N Thompson
Jan 1, 1985·Journal of Inherited Metabolic Disease·A Tylki-SzymańskaB Czartoryska
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Feb 20, 2020·International Journal of Molecular Sciences·Francesca D'AvanzoRosella Tomanin
Jun 1, 1984·Developmental Medicine and Child Neurology·M Adinolfi, S Brown
Jan 1, 1993·Developmental Medicine and Child Neurology·M Adinolfi

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