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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: muntoni f. 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.
T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy.
Anthony K, Ala P, Catapano F, Meng J, Domingos J, Perry M, Ricotti V, Maresh K, Phillips LC, Servais L, Seferian AM, De Lucia S, de Groot I, Krom YD, Verschuuren JGM, Niks EH, Straub V, Guglieri M, Voit T, Morgan J, Muntoni F. Anthony K, et al. Among authors: muntoni f. Hum Gene Ther. 2023 May;34(9-10):439-448. doi: 10.1089/hum.2022.166. Epub 2023 Feb 24. Hum Gene Ther. 2023. PMID: 36453228
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice.
Saoudi A, Barberat S, le Coz O, Vacca O, Doisy Caquant M, Tensorer T, Sliwinski E, Garcia L, Muntoni F, Vaillend C, Goyenvalle A. Saoudi A, et al. Among authors: muntoni f. Mol Ther Nucleic Acids. 2023 Mar 21;32:173-188. doi: 10.1016/j.omtn.2023.03.009. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37078061 Free PMC article.
Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model.
Doisy M, Vacca O, Fergus C, Gileadi T, Verhaeg M, Saoudi A, Tensorer T, Garcia L, Kelly VP, Montanaro F, Morgan JE, van Putten M, Aartsma-Rus A, Vaillend C, Muntoni F, Goyenvalle A. Doisy M, et al. Among authors: muntoni f. Biomedicines. 2023 Dec 7;11(12):3243. doi: 10.3390/biomedicines11123243. Biomedicines. 2023. PMID: 38137463 Free PMC article.
Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping.
Mercuri E, Seferian AM, Servais L, Deconinck N, Stevenson H, Ni X, Zhang W, East L, Yonren S, Muntoni F; 4658-102 Study Group. Mercuri E, et al. Among authors: muntoni f. Neuromuscul Disord. 2023 Jun;33(6):476-483. doi: 10.1016/j.nmd.2023.03.008. Epub 2023 Mar 24. Neuromuscul Disord. 2023. PMID: 37207382 Free article.
925 results