A therapeutic trial of amniotic epithelial cell implantation in patients with lysosomal storage diseases
作者： Andrew M. Yeager , Harvey S. Singer , James R. Buck , Reuben Matalon , Susan Brennan
关键词: Leukodystrophy 、 In patient 、 Pathology 、 Gangliosidoses 、 Progressive disease 、 Amnion 、 Allotransplantation 、 Clinical trial 、 Biology 、 Immunology 、 Epithelium
摘要: To determine whether allografts of normal amniotic epithelium might provide a nonimmunogenic cellular source exogenous lysosomal enzymes, subcutaneous implants were performed in six children with clinically advanced storage diseases. The clinical and the biochemical status each patient was observed for several weeks after epithelial cell implantation (AECI). Serial studies blood samples from post-AECI period did not demonstrate any increase levels deficient hydrolase. In two patients, quantitative urinary excretion substrate also studied show consistent alterations AECI. No had objective improvement or neurodevelopmental following Two patients died progressive disease at 2 1/2 3 mo AECI; no residual found postmortem examination. Four are alive 6-14 We conclude that human amnion do sufficient replacement hydrolases
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