Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/...
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
About this item
Full title
Author / Creator
Russell, Stephen R. , Drack, Arlene V. , Cideciyan, Artur V. , Jacobson, Samuel G. , Leroy, Bart P. , 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 and Girach, Aniz
Publisher
New York: Nature Publishing Group US
Journal title
Language
English
Formats
Publication information
Publisher
New York: Nature Publishing Group US
Subjects
More information
Scope and Contents
Contents
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 t...
Alternative Titles
Full title
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
Authors, Artists and Contributors
Author / Creator
Drack, Arlene V.
Cideciyan, Artur V.
Jacobson, Samuel G.
Leroy, Bart P.
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_9117145
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_9117145
Other Identifiers
ISSN
1078-8956
E-ISSN
1546-170X
DOI
10.1038/s41591-022-01755-w
