Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease

Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease

Abstract Chronic granulomatous disease (CGD) is a severe inborn error of immunity caused by NADPH oxidase defects. Here, we develop CRISPR/Cas9-based gene editing strategies for correction of variants in the CYBA and CYBB genes causing CGD. For X-linked CGD, we also develop a near-universal gene edi...

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Main Authors: Jonas Holst Wolff, Thomas Wisbech Skov, Didde Haslund, Sofie Rahbek Dorset, Anne Louise S. Revenfeld, Clotilde Aussel, Sofie E. Jørgensen, Mette Holm, Martin K. Thomsen, Sandra Ammann, Toni Cathomen, Trine H. Mogensen, Bjarne Kuno Møller, Rasmus O. Bak, Jacob Giehm Mikkelsen
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-62738-2
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