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CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing.
van Agtmaal EL, André LM, Willemse M, Cumming SA, van Kessel IDG, van den Broek WJAA, Gourdon G, Furling D, Mouly V, Monckton DG, Wansink DG, Wieringa B. van Agtmaal EL, et al. Among authors: willemse m. Mol Ther. 2017 Jan 4;25(1):24-43. doi: 10.1016/j.ymthe.2016.10.014. Epub 2017 Jan 4. Mol Ther. 2017. PMID: 28129118 Free PMC article.
The nuclear concentration required for antisense oligonucleotide activity in myotonic dystrophy cells.
van der Bent ML, Paulino da Silva Filho O, Willemse M, Hällbrink M, Wansink DG, Brock R. van der Bent ML, et al. Among authors: willemse m. FASEB J. 2019 Oct;33(10):11314-11325. doi: 10.1096/fj.201900263R. Epub 2019 Jul 16. FASEB J. 2019. PMID: 31311315
Our findings have important implications for the clinical use of ASOs in DM1 and provide a basis for further research on other types of ASOs.-Van der Bent, M. L., Paulino da Silva Filho, O., Willemse, M., Hallbrink, M., Wansink, D. G., Brock, R. ...
Our findings have important implications for the clinical use of ASOs in DM1 and provide a basis for further research on other types of ASOs …
76 results