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Molecular Therapy. Nucleic Acids|August 4, 2023
The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disordersXiang Li, Mahboubeh Kheirabadi, Patrick G Dougherty, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|May 21, 2026
Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophyEmma N Shea, Hailey R Olafson, Derek R Muscato, et al.Pageof 2