Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1
| Autor: | El Boujnouni, Najoua, van der Bent, M. Leontien, Willemse, Marieke, ’t Hoen, Peter A.C., Brock, Roland, Wansink, Derick G. |
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| Zdroj: | In Molecular Therapy - Nucleic Acids 13 June 2023 32:622-636 |
| Databáze: | ScienceDirect |
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