Search Results - "den Hollander, Wilhelmina"

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

“…CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease resulting in childhood blindness. Sepofarsen is an RNA antisense…”
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Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis by Drevinek, Pavel, Pressler, Tacjana, Cipolli, Marco, De Boeck, Kris, Schwarz, Carsten, Bouisset, Florilene, Boff, Marie, Henig, Noreen, Paquette-Lamontagne, Nicolas, Montgomery, Sonya, Perquin, Jaakko, Tomkinson, Nigel, den Hollander, Wilhelmina, Elborn, J. Stuart

“…Eluforsen is an antisense oligonucleotide designed to bind to the mRNA region around the F508-encoding deletion and restore the cystic fibrosis transmembrane…”
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Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis by Sermet-Gaudelus, Isabelle, Clancy, John P., Nichols, David P., Nick, Jerry A., De Boeck, Kris, Solomon, George M., Mall, Marcus A., Bolognese, James, Bouisset, Florilene, den Hollander, Wilhelmina, Paquette-Lamontagne, Nicolas, Tomkinson, Nigel, Henig, Noreen, Elborn, J. Stuart, Rowe, Steven M.

“…Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. In this study we assessed the effect of antisense…”
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