Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10 : a phase 1b/2 trial

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10 : a phase 1b/2 trial

CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease resulting in childhood blindness. Sepofarsen is an RNA antisense oligonucleotide targeting the c.2991+1655A>G variant in the CEP290 gene to treat LCA10. In this open-label, phase 1b/2 (NCT03140969), 12-month, multic...

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Hauptverfasser: Russell, Stephen R, Drack, Arlene V, Cideciyan, Artur V, Jacobson, Samuel G, Leroy, Bart, Van Cauwenbergh, Caroline, Ho, Allen C, Dumitrescu, Alina V, Han, Ian C, Martin, Mitchell, Pfeifer, Wanda L, Sohn, Elliott H, Walshire, Jean, Garafalo, Alexandra V, Krishnan, Arun K, Powers, Christian A, Sumaroka, Alexander, Roman, Alejandro J, Vanhonsebrouck, Eva, Jones, Eltanara, Nerinckx, Fanny, De Zaeytijd, Julie, Collin, Rob W. J, Hoyng, Carel, Adamson, Peter, Cheetham, Michael E, Schwartz, Michael R, den Hollander, Wilhelmina, Asmus, Friedrich, Platenburg, Gerard, Rodman, David, Girach, Aniz
Format: Artikel
Sprache:eng
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General Biochemistry, Genetics and Molecular Biology
General Medicine
Medicine and Health Sciences
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Zusammenfassung:CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease resulting in childhood blindness. Sepofarsen is an RNA antisense oligonucleotide targeting the c.2991+1655A>G variant in the CEP290 gene to treat LCA10. In this open-label, phase 1b/2 (NCT03140969), 12-month, multicenter, multiple-dose, dose-escalation trial, six adult patients and five pediatric patients received
ISSN:1078-8956
1546-170X