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• W2040355286 abstract "Enzyme replacement therapy (ERT) for the lysosomal storage disease mucopolysaccharidosis I (MPS I) involves i.v. injection of alpha-l-iduronidase, which can be taken up by cells throughout the body. While a significant immune response to ERT has been shown in patients with MPS I, little is known about what effect anti-enzyme antibodies have on treatment efficacy. In this issue of the JCI, Dickson et al. demonstrate that anti-enzyme antibodies inhibit enzyme uptake and substantially limit the therapeutic efficacy of ERT in canines with MPS I (see the related article beginning on page 2868). Furthermore, the induction of immune tolerance--via oral delivery of cyclosporine A and azathioprine for two months at the time of initiation of ERT with recombinant human alpha-L-iduronidase--improved enzyme uptake in organs. Therefore, transient immunosuppression may enhance ERT for lysosomal storage diseases." @default.
• W2040355286 created "2016-06-24" @default.
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• W2040355286 date "2008-07-24" @default.
• W2040355286 modified "2023-09-24" @default.
• W2040355286 title "Immune response hinders therapy for lysosomal storage diseases" @default.
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• W2040355286 doi "https://doi.org/10.1172/jci36521" @default.
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