Φορτώνει......
Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction
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...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Mol Ther Methods Clin Dev |
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| Κύριοι συγγραφείς: | , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
American Society of Gene & Cell Therapy
2017
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5363303/ https://ncbi.nlm.nih.gov/pubmed/28344998 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2016.12.010 |
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