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CRISPR-mediated correction of skeletal muscle Ca2+ handling in a novel DMD patient-derived pluripotent stem cell model.
Morera C, Kim J, Paredes-Redondo A, Nobles M, Rybin D, Moccia R, Kowala A, Meng J, Garren S, Liu P, Morgan JE, Muntoni F, Christoforou N, Owens J, Tinker A, Lin YY. Morera C, et al. Among authors: paredes redondo a. Neuromuscul Disord. 2022 Dec;32(11-12):908-922. doi: 10.1016/j.nmd.2022.10.007. Epub 2022 Nov 1. Neuromuscul Disord. 2022. PMID: 36418198 Free article.
A new patient-derived iPSC model for dystroglycanopathies validates a compound that increases glycosylation of α-dystroglycan.
Kim J, Lana B, Torelli S, Ryan D, Catapano F, Ala P, Luft C, Stevens E, Konstantinidis E, Louzada S, Fu B, Paredes-Redondo A, Chan AE, Yang F, Stemple DL, Liu P, Ketteler R, Selwood DL, Muntoni F, Lin YY. Kim J, et al. Among authors: paredes redondo a. EMBO Rep. 2019 Nov 5;20(11):e47967. doi: 10.15252/embr.201947967. Epub 2019 Sep 30. EMBO Rep. 2019. PMID: 31566294 Free PMC article.