A CRISPR/Cas9 screen reveals proteins at the endosome-Golgi interface that modulate cellular anti-sense oligonucleotide activity

A CRISPR/Cas9 screen reveals proteins at the endosome-Golgi interface that modulate cellular anti-sense oligonucleotide activity

Abstract Anti-sense oligonucleotides (ASOs) are modified synthetic single-stranded molecules with enhanced stability, activity, and bioavailability. They associate with RNA through sequence complementarity and can reduce or alter mRNA expression upon binding of splice site positions. To target RNA i...

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Main Authors: Liza Malong, Jessica Roskosch, Carolina Hager, Jean-Philippe Fortin, Roland Schmucki, Marinella G. Callow, Christian Weile, Valentina Romeo, Christoph Patsch, Scott Martin, Mike Costa, Zora Modrusan, Roberto Villaseñor, Erich Koller, Benjamin Haley, Anne Spang, Filip Roudnicky
Format: Article
Language:English
Published: Nature Portfolio 2025-06-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-61039-y
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https://doi.org/10.1038/s41467-025-61039-y

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