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Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells.
Porquet F, Weidong L, Jehasse K, Gazon H, Kondili M, Blacher S, Massotte L, Di Valentin E, Furling D, Gillet NA, Klein AF, Seutin V, Willems L. Porquet F, et al. Among authors: klein af. Mol Ther Nucleic Acids. 2023 May 13;32:857-871. doi: 10.1016/j.omtn.2023.05.007. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37273786 Free PMC article.
Cells of Matter-In Vitro Models for Myotonic Dystrophy.
Matloka M, Klein AF, Rau F, Furling D. Matloka M, et al. Among authors: klein af. Front Neurol. 2018 May 23;9:361. doi: 10.3389/fneur.2018.00361. eCollection 2018. Front Neurol. 2018. PMID: 29875732 Free PMC article. Review.
FISH Protocol for Myotonic Dystrophy Type 1 Cells.
Klein AF, Arandel L, Marie J, Furling D. Klein AF, et al. Methods Mol Biol. 2020;2056:203-215. doi: 10.1007/978-1-4939-9784-8_13. Methods Mol Biol. 2020. PMID: 31586350
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells.
Dastidar S, Ardui S, Singh K, Majumdar D, Nair N, Fu Y, Reyon D, Samara E, Gerli MFM, Klein AF, De Schrijver W, Tipanee J, Seneca S, Tulalamba W, Wang H, Chai YC, In't Veld P, Furling D, Tedesco FS, Vermeesch JR, Joung JK, Chuah MK, VandenDriessche T. Dastidar S, et al. Among authors: klein af. Nucleic Acids Res. 2018 Sep 19;46(16):8275-8298. doi: 10.1093/nar/gky548. Nucleic Acids Res. 2018. PMID: 29947794 Free PMC article.
Reversal of RNA toxicity in myotonic dystrophy via a decoy RNA-binding protein with high affinity for expanded CUG repeats.
Arandel L, Matloka M, Klein AF, Rau F, Sureau A, Ney M, Cordier A, Kondili M, Polay-Espinoza M, Naouar N, Ferry A, Lemaitre M, Begard S, Colin M, Lamarre C, Tran H, Buée L, Marie J, Sergeant N, Furling D. Arandel L, et al. Among authors: klein af. Nat Biomed Eng. 2022 Feb;6(2):207-220. doi: 10.1038/s41551-021-00838-2. Epub 2022 Feb 10. Nat Biomed Eng. 2022. PMID: 35145256
26 results