Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I

Enzyme replacement therapy with laronidase is an established treatment for Mucopolysaccharidosis type I (MPS I), but its efficacy may be limited by the development of anti-drug antibodies, which inhibit cellular uptake of the enzyme. In a related disorder, infantile Pompe disease, immune tolerance i...

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Bibliografiska uppgifter
I publikationen:Mol Ther Methods Clin Dev
Huvudupphovsmän: Ghosh, Arunabha, Liao, Aiyin, O’Leary, Claire, Mercer, Jean, Tylee, Karen, Goenka, Anu, Holley, Rebecca, Jones, Simon A., Bigger, Brian W.
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2019
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC6441787/
https://ncbi.nlm.nih.gov/pubmed/30976609
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.02.007
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