Successful therapy of Niemann-Pick disease by implantation of human amniotic membrane.
In a patient with a lysosomal storage disorder, not involving the CNS, repeated implantations of human amniotic sheets have proved to provide a successful approach to enzyme replacement therapy. Implantation of pure epithelial cells, separated from the other cell types of the amnion, might markedly improve the procedure, avoiding some risks of host-versus-graft rejection.
D000293:Adolescent; D000650:Amnion / Q000201:enzymology / Q000637:transplantation*; D004848:Epithelium / Q000201:enzymology / Q000637:transplantation; D005260:Female; D006085:Graft Survival; D006789:Host vs Graft Reaction; D006801:Humans; D008297:Male; D009542:Niemann-Pick Diseases / Q000473:pathology / Q000628:therapy*; D010727:Phosphoric Diester Hydrolases / Q000172:deficiency*; D011247:Pregnancy; D013108:Sphingomyelin Phosphodiesterase / Q000172:deficiency*
D010727:Phosphoric Diester Hydrolases; D013108:Sphingomyelin Phosphodiesterase