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Targeted knock-in of a NCF1 coding sequence into the endogenous NCF2 locus leads to myeloid phenotypic correction of p47 -deficient chronic granulomatous disease

Kah Mun Siow, Merve Güngör,Dominik Wrona,Federica Raimondi,Oleksandr Pastukhov,Panagiotis Tsapogas, Timon Menzi,Michael Schmitz, Péter István Kulcsár,Gerald Schwank,Ansgar Schulz, Martin Jinek,Ute Modlich,Ulrich Siler,Janine Reichenbach

Molecular Therapy - Nucleic Acids（2024）

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