Utility of a 3D–Engineered Skeletal Muscle Organoid System to Assess Exon Skipping, Dystrophin Protein Restoration, and Functional Improvement in a Human DMD Cell Model

Author(s): Nelsa L. Estrella, Sara L. Blake, Holly McKee, Maureen Fredericks, Christopher M. Brennan, Matthew Streeter, Amanda Dombroski, George Appiah Kubi, Ning Li, Pressley Smith, Shalimar Hachem, Ruyuan Wei, Amy N. Hicks, Mahboubeh Kheirabadi, Mark A. Wysk, Ningguo Gao, Ziqing Leo Qian, Muralidhar Mallem, Mahasweta Girgenrath