Exon-targeted U1 snRNA rescues splicing defects and ameliorates inherited optic neuropathy: A novel RNA-based treatment
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Authors
- Zhengyang Chen (ORCID: https://orcid.org/0009-0004-7340-4150)
- Zhiyong Hu (ORCID: https://orcid.org/0000-0002-9082-3056)
- Xiaoyan Sun
- Wei Chen
- Yuting Li
Institutions
- Shanghai Jiao Tong University (CN)
- Fudan University (CN)
- Shanghai Sixth People's Hospital (CN)
Publication Details
- Journal
- Molecular Therapy — Nucleic Acids
- Published
- 2026-02-25
- DOI
- https://doi.org/10.1016/j.omtn.2026.102868
- Primary Topic
- RNA Research and Splicing
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Natural Science Foundation of China