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...

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Detalles Bibliográficos
Autores principales: Cideciyan, Artur V., Jacobson, Samuel G., Ho, Allen C., Swider, Malgorzata, Sumaroka, Alexander, Roman, Alejandro J., Wu, Vivian, Russell, Robert C., Viarbitskaya, Iryna, Garafalo, Alexandra V., Schwartz, Michael R., Girach, Aniz
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10302566/
https://www.ncbi.nlm.nih.gov/pubmed/37388818
http://dx.doi.org/10.1016/j.ajoc.2023.101873

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10302566/
https://www.ncbi.nlm.nih.gov/pubmed/37388818
http://dx.doi.org/10.1016/j.ajoc.2023.101873

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