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
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将NCF1编码序列靶向敲入内源性NCF2基因座可使p47缺陷型慢性肉芽肿病的髓样表型得到纠正
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