Cargando…
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...
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: | |
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 |
Ejemplares similares
-
Treatment Potential for Macular Cone Vision in Leber Congenital Amaurosis Due to CEP290 or NPHP5 Mutations: Predictions From Artificial Intelligence
por: Sumaroka, Alexander, et al.
Publicado: (2019) -
Alternative splicing in CEP290 mutant cats results in a milder phenotype than LCA
(CEP290)
patients
por: Minella, Andrea L., et al.
Publicado: (2022) -
Genetic Screening of LCA in Belgium: Predominance of CEP290 and Identification of Potential Modifier Alleles in AHI1 of CEP290-related Phenotypes
por: Coppieters, Frauke, et al.
Publicado: (2010) -
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
por: Russell, Stephen R., et al.
Publicado: (2022) -
LEBER CONGENITAL AMAUROSIS DUE TO CEP290 MUTATIONS—SEVERE VISION IMPAIRMENT WITH A HIGH UNMET MEDICAL NEED: A Review
por: Leroy, Bart P., et al.
Publicado: (2021)