Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease

Babies born with Pompe disease require life-long treatment with enzyme-replacement therapy (ERT). Despite the human origin of the therapy, recombinant human lysosomal acid α glucosidase (GAA, rhGAA), ERT unfortunately leads to the development of high titers of anti-rhGAA antibody, decreased effectiv...

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Hlavní autoři: Cousens, Leslie P., Mingozzi, Federico, van der Marel, Sander, Su, Yan, Garman, Richard, Ferreira, Valerie, Martin, William, Scott, David W., De Groot, Anne S.
Médium: Artigo
Jazyk:Inglês
Vydáno: Landes Bioscience 2012
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3660767/
https://ncbi.nlm.nih.gov/pubmed/23095864
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4161/hv.21405
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