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Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

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Pompe disease results from acid α-glucosidase (GAA) deficiency, and enzyme replacement therapy (ERT) with recombinant human (rh) GAA has clinical benefits, although its limitations include the short half-life of GAA and the formation of antibody responses. The present study compared the efficacy of...

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Detalles Bibliográficos
Autores principales:	Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363303/ https://www.ncbi.nlm.nih.gov/pubmed/28344998 http://dx.doi.org/10.1016/j.omtm.2016.12.010

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