280. Combination of Low-Dose Gene Therapy and Monthly Enzyme Replacement Therapy Improves the Phenotype of a Mouse Model of Lysosomal Storage Disease
| Autor: | Ferla, Rita 1, 2, Alliegro, Marialuisa 1, 2, Nusco, Edoardo 1, Claudiani, Pamela 1, 2, Auricchio, Alberto 1, 2 |
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| Zdroj: | In Molecular Therapy May 2015 23 Supplement 1:S112-S112 |
| Databáze: | ScienceDirect |
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