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TREATMENT OF CHRONIC GRANULOMATOUS DISEASE

机译:治疗慢性肉芽肿病

摘要

The present invention relates to an isolated human hematopoietic stem cell or progenitor cell, transduced with a lentiviral vector which comprises a coding nucleic acid sequence encoding a functional variant of a polypeptide selected from gp91phox, p22phox, p40phox, p47phox, p67phox and Rac2; under transcriptional control of a promoter sequence that comprises or essentially consists of the miR223 promoter sequence (SEQ ID NO 01).
机译:本发明涉及分离的人造血干细胞或祖细胞,其用慢病毒载体转导,所述慢病毒载体包括编码选自GP91phox,P22phox,P40KON,P47phox,P67phox和Rac2的多肽的官能变体的编码核酸序列;在包含或基本上由MiR223启动子序列(SEQ ID NO 01)组成的启动子序列的转录控制。

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