A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease

A major obstacle to enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) for Pompe disease is the development of high titers of anti-rhGAA antibodies in a subset of patients, which often leads to a loss of treatment efficacy. In an effort to induce sustained immune tole...

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Detalles Bibliográficos
Publicado en:Mol Genet Metab Rep
Autores principales: Lim, Han-Hyuk, Yi, Haiqing, Kishimoto, Takashi K., Gao, Fengqin, Sun, Baodong, Kishnani, Priya S.
Formato: Artigo
Lenguaje:Inglês
Publicado: Elsevier 2017
Materias:
Research Paper
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5524423/
https://ncbi.nlm.nih.gov/pubmed/28761815
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.03.005
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