Restoration of Cone Sensitivity to Individuals with Congenital Photoreceptor Blindness within the Phase 1/2 Sepofarsen Trial

PURPOSE: To understand consequences of reconstituting cone photoreceptor function in congenital binocular blindness resulting from mutations in the centrosomal protein 290 (CEP290) gene. DESIGN: Phase 1b/2 open-label, multicenter, multiple-dose, dose-escalation trial. PARTICIPANTS: A homogeneous sub...

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Detalles Bibliográficos
Autores principales: Cideciyan, Artur V., Jacobson, Samuel G., Ho, Allen C., Krishnan, Arun K., Roman, Alejandro J., Garafalo, Alexandra V., Wu, Vivian, Swider, Malgorzata, Sumaroka, Alexander, Van Cauwenbergh, Caroline, Russell, Stephen R., Drack, Arlene V., Leroy, Bart P., Schwartz, Michael R., Girach, Aniz
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9562351/
https://www.ncbi.nlm.nih.gov/pubmed/36249682
http://dx.doi.org/10.1016/j.xops.2022.100133

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9562351/
https://www.ncbi.nlm.nih.gov/pubmed/36249682
http://dx.doi.org/10.1016/j.xops.2022.100133

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