Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes
Purpose: An intravitreally injected antisense oligonucleotide, sepofarsen, was designed to modulate splicing within retinas of patients with severe vision loss due to deep intronic c.2991 + 1655A > G variant in the CEP290 gene. A previous report showed vision improvements following a single injec...
| 主要な著者: | , , , , , , , , , , , |
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| フォーマット: | 論文 |
| 言語: | English |
| 出版事項: |
Elsevier
2023-12-01
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| シリーズ: | American Journal of Ophthalmology Case Reports |
| 主題: | |
| オンライン･アクセス: | http://www.sciencedirect.com/science/article/pii/S2451993623000816 |