Investigative ophthalmology & visual science۲۰۲۵۲ استنادنمایه شده در PubMed
Wuyi Li, Yamei Li, Yunyu Zhou, Yue Liu, Huixin Liu, Xing Wei, Zixi Sun, Xiaoxu Han, Xuan Zou, Hui Li, Ruifang Sui
چکیده
This study aimed to evaluate the exon-skipping efficacy and safety of an antisense oligonucleotide (AON) targeting USH2A exon 13 across multiple models, including the Rb1 cell line, humanized USH2A-e13 transgenic mice, and patient-derived retinal organoids. Additionally, we investigated the…
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DOI: 10.1167/iovs.66.12.46