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Exon-targeted U1 snRNA rescues splicing defects and ameliorates inherited optic neuropathy: A novel RNA-based treatment.

Hongli Zhang, Xiaoyan Sun, Zhengyang Chen, Yuting Li, Wei Chen

2026.3.1Molecular Therapy Nucleic Acids

DOI: 10.1016/j.omtn.2026.102868

Abstract

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Citation format

ZHANG, Hongli, et al. Exon-targeted u1 snrna rescues splicing defects and ameliorates inherited optic neuropathy: A novel RNA-based treatment. Molecular Therapy Nucleic Acids, 2026, 37 1(1): 102868.