caa a22 4500 469043806 CHVBK 20180323132818.0 cr unu---uuuuu 170328e19920101xx s 000 0 eng 10.1007/BF01800340 doi (NATIONALLICENCE)springer-10.1007/BF01800340 Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses [Elektronische Daten] [J. Muenzer, E. Neufeld, G. Constantopoulos, R. Caruso, M. Kaiser-Kupfer, A. Pikus, J. Danoff, R. Berry, H. McDonald, J. Thompson, L. Rodén, M. Zasloff] Summary: Amnion membrane implantation has been proposed as an approach to enzyme replacement in mucopolysaccharidoses. Human amnion membranes have been subcutaneously implanted in the abdominal wall in 19 patients with mucopolysaccharidoses (MPS I, II and III). A protocol was developed for the objective evaluation of experimental treatments of these patients. Systematic evaluation of the clinical status before and 6 months after amnion membrane implantation reveals no change in function except improvement in joint mobility. The sum of all joint movements showed improvement from baseline values to 6 months after implantation by ANOVA followed bypost-hoc analysis (p<0.056). The only specific joint movements to significantly improve after 6 months were shoulder extension (p<0.01) and hip internal rotation (p<0.05). Serial measurements of the deficient lysosomal enzyme activity in serum and white blood cells did not increase in any patient after amnion membrane implantation. Urinary glycosaminoglycan excretion decreased transiently in 2 of 10 patients after implantation, but a second amnion membrane implantation did not result in any change. Biopsy of the implantation site in 10 patients 6 months after amnion membrane implantation revealed a foreign-body reaction with giant cell formation and fibrosis and no recognizable amnion membrane tissue. We conclude that human amnion membrane implantation is not an effective therapy in mucopolysaccharidoses. SSIEM and Kluwer Academic Publishers, 1992 Muenzer J. Human Genetics Branch, National Institute of Child Health and Human Development, USA aut Neufeld E. Genetics and Biochemistry Branch, National Institute of Diabetes, Digestive and Kidney Diseases, USA aut Constantopoulos G. Developmental and Metabolic Neurology Branch, National Institute of Neurological and Communicative Disorders and Stroke, USA aut Caruso R. Ophthalmic Genetics and Clinical Services Branch, National Eye Institute, USA aut Kaiser-Kupfer M. Ophthalmic Genetics and Clinical Services Branch, National Eye Institute, USA aut Pikus A. Audiology, USA aut Danoff J. Rehabilitation Medicine Departments, Warren Grant Magnuson Clinical Center, National Institutes of Health, USA aut Berry R. Rehabilitation Medicine Departments, Warren Grant Magnuson Clinical Center, National Institutes of Health, USA aut McDonald H. Surgery Branch, Division of Cancer Treatment, National Cancer Institute, National Institutes of Health, 20892, Bethesda, MD, USA aut Thompson J. Laboratory of Medical Genetics, University of Alabama, 35294, Birmingham, AL, USA aut Rodén L. Laboratory of Medical Genetics, University of Alabama, 35294, Birmingham, AL, USA aut Zasloff M. Human Genetics Branch, National Institute of Child Health and Human Development, USA aut Journal of Inherited Metabolic Disease Kluwer Academic Publishers 15/1(1992-01-01), 25-37 0141-8955 15:1<25 1992 15 10545 https://doi.org/10.1007/BF01800340 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/BF01800340 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Muenzer J. Human Genetics Branch, National Institute of Child Health and Human Development, USA aut NATIONALLICENCE 700 1- Neufeld E. Genetics and Biochemistry Branch, National Institute of Diabetes, Digestive and Kidney Diseases, USA aut NATIONALLICENCE 700 1- Constantopoulos G. Developmental and Metabolic Neurology Branch, National Institute of Neurological and Communicative Disorders and Stroke, USA aut NATIONALLICENCE 700 1- Caruso R. Ophthalmic Genetics and Clinical Services Branch, National Eye Institute, USA aut NATIONALLICENCE 700 1- Kaiser-Kupfer M. Ophthalmic Genetics and Clinical Services Branch, National Eye Institute, USA aut NATIONALLICENCE 700 1- Pikus A. Audiology, USA aut NATIONALLICENCE 700 1- Danoff J. Rehabilitation Medicine Departments, Warren Grant Magnuson Clinical Center, National Institutes of Health, USA aut NATIONALLICENCE 700 1- Berry R. Rehabilitation Medicine Departments, Warren Grant Magnuson Clinical Center, National Institutes of Health, USA aut NATIONALLICENCE 700 1- McDonald H. Surgery Branch, Division of Cancer Treatment, National Cancer Institute, National Institutes of Health, 20892, Bethesda, MD, USA aut NATIONALLICENCE 700 1- Thompson J. Laboratory of Medical Genetics, University of Alabama, 35294, Birmingham, AL, USA aut NATIONALLICENCE 700 1- Rodén L. Laboratory of Medical Genetics, University of Alabama, 35294, Birmingham, AL, USA aut NATIONALLICENCE 700 1- Zasloff M. Human Genetics Branch, National Institute of Child Health and Human Development, USA aut NATIONALLICENCE 773 0- Journal of Inherited Metabolic Disease Kluwer Academic Publishers 15/1(1992-01-01), 25-37 0141-8955 15:1<25 1992 15 10545 Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer