Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy

Molecular Therapy 2026 Open access

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Authors: Emma N. Shea, Hailey R. Olafson, Derek R. Muscato, Leanne M. Adams, M. Carmen Valero, Kendra McKee, Xiulong Shen, Patrick G. Dougherty, Amy N. Hicks, Nanjun Liu, Mahboubeh Kheirabadi, Matthew Streeter
+6 more Xiang Li, Phallika Mon, Mahasweta Girgenrath, Natarajan Sethuraman, Ziqing Qian, Eric T. Wang
DOI: 10.1016/j.ymthe.2026.04.060
Cited by: 1
References: 33

Fields of Study & Subjects

Genetic Neurodegenerative Diseases Amyotrophic Lateral Sclerosis Research RNA Research and Splicing

Publication details

Journal / source
Molecular Therapy
Publisher
Elsevier BV
Volume
34
Issue
8
Pages
4844–4855
Publication date
19 May 2026
Language
EN