AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes
| Autor: | Keskin, Sonay, Brouwers, Cynthia C., Sogorb-Gonzalez, Marina, Martier, Raygene, Depla, Josse A., Vallès, Astrid, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M. |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 13 December 2019 15:275-284 |
| Databáze: | ScienceDirect |
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