Publication: Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 $^{phox}$ -deficient chronic granulomatous disease
Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 $^{phox}$ -deficient chronic granulomatous disease
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Siow, K. M., Güngör, M., Wrona, D., Raimondi, F., Pastukhov, O., Tsapogas, P., Menzi, T., Schmitz, M., Kulcsár, P. I., Schwank, G., Schulz, A., Jinek, M., Modlich, U., Siler, U., & Reichenbach, J. (2024). Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 $^{phox}$ -deficient chronic granulomatous disease. Molecular Therapy : Nucleic Acids, 35, 102229. https://doi.org/10.1016/j.omtn.2024.102229
Abstract
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Abstract
p47 $^{phox}$ -deficient chronic granulomatous disease (p47-CGD) is a primary immunodeficiency caused by mutations in the neutrophil cytosolic factor 1 (NCF1) gene, resulting in defective NADPH oxidase function in phagocytes. Due to its complex genomic context, the NCF1 locus is not suited for safe gene editing with current genome editing technologies. Therefore, we developed a targeted NCF1 coding sequence knock-in by CRISPR-Cas9 ribonucleoprotein and viral vector template delivery, to restore p47 $^{phox}$
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- Siow, Kah Mun
- Güngör, Merve
- Wrona, Dominik
- Raimondi, Federica
- Pastukhov, Oleksandr
- Tsapogas, Panagiotis
- Menzi, Timon
- Schmitz, Michael
- Kulcsár, Péter István
- Schwank, Gerald
- Schulz, Ansgar
- Jinek, Martin
- Modlich, Ute
- Siler, Ulrich
- Reichenbach, Janine
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Siow, K. M., Güngör, M., Wrona, D., Raimondi, F., Pastukhov, O., Tsapogas, P., Menzi, T., Schmitz, M., Kulcsár, P. I., Schwank, G., Schulz, A., Jinek, M., Modlich, U., Siler, U., & Reichenbach, J. (2024). Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 $^{phox}$ -deficient chronic granulomatous disease. Molecular Therapy : Nucleic Acids, 35, 102229. https://doi.org/10.1016/j.omtn.2024.102229
