Treatment of chronic granulomatous disease
UA131274C2Undetermined Publication Date: 2026-10-07UNIVERSITY OF ZURICH
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Patent Information
- Authority / Receiving Office
- UA · UA
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2020-10-02
- Filing Date
- 2020-10-02
- Publication Date
- 2026-10-07
Abstract
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).
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