Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

Infantile Pompe disease progresses to a lethal cardiomyopathy in absence of effective treatment. Enzyme-replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) has been effective in most patients with Pompe disease, but efficacy was reduced by high-titer antibody responses. Immun...

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Hlavní autoři: Sun, Baodong, Kulis, Michael D, Young, Sarah P, Hobeika, Amy C, Li, Songtao, Bird, Andrew, Zhang, Haoyue, Li, Yifan, Clay, Timothy M, Burks, Wesley, Kishnani, Priya S, Koeberl, Dwight D
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2009
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Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2818301/
https://ncbi.nlm.nih.gov/pubmed/19690517
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.195
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