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Disease modeling and gene correction of LGMDR21 iPSCs elucidates the role of POGLUT1 in skeletal muscle maintenance, regeneration, and the satellite cell niche.
Ortiz-Vitali JL, Wu J, Xu N, Shieh AW, Niknejad N, Takeuchi M, Paradas C, Lin C, Jafar-Nejad H, Haltiwanger RS, Wang SH, Darabi R. Ortiz-Vitali JL, et al. Mol Ther Nucleic Acids. 2023 Aug 2;33:683-697. doi: 10.1016/j.omtn.2023.07.037. eCollection 2023 Sep 12. Mol Ther Nucleic Acids. 2023. PMID: 37650119 Free PMC article.