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在CEP290-LCA中使用反感性寡核酸进行单次视内治疗后,持续的视力改善:在两只眼睛中复制
Artur V Cideciyan1, Samuel G Jacobson1, Allen C Ho2
1Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
American journal of ophthalmology case reports
|June 30, 2023
概括
塞波法森为CEP290相关视力损失的患者提供了持久的视力改善. 每次注射后,疗效持续了大约两年,这表明更长的再治疗间隔可能是有益的.
科学领域:
- 眼科医生 眼科 眼科
- 遗传医学是一种遗传医学.
- 分子生物学分子生物学
背景情况:
- 塞波法森 (Sepofarsen) 是一种反感性寡核酸,它针对CEP290基因中的深层内基因c.2991+1655A>G变体.
- 这种变异在患有遗传性视网膜疾病的患者中导致严重的视力丧失.
- 之前的研究表明,单次注射sepofarsen后,视力持续改善.
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