Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction
| Autor: | Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 17 March 2017 4:126-136 |
| Databáze: | ScienceDirect |
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