321. Deletion of Mutated GAA Repeats from the Intron 1 of the Frataxin Gene Using the CRISPR System Restores the Protein Expression in a Friedreich Ataxia Model
| Autor: | Ouellet, Dominique L., Rousseau, Joel, Cherif, Khadija, Gérard, Catherine, Gilbert, Renald, Garnier, Alain, Gaillet, Bruno, Beauséjour, Christian, Tremblay, Jacques P. |
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| Zdroj: | In Molecular Therapy May 2016 24 Supplement 1:S129-S129 |
| Databáze: | ScienceDirect |
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