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HAEMATOPOIETIC STEM CELL-GENE THERAPY FOR WISKOTT-ALDRICH SYNDROME
HAEMATOPOIETIC STEM CELL-GENE THERAPY FOR WISKOTT-ALDRICH SYNDROME
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机译:Wiskott-Aldrich综合征的出血毒性干细胞基因治疗
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摘要
The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a Wiskott-Aldrich Syndrome protein. In some embodiments, the expression vector is a self-inactivating lentiviral vector. In some embodiments, the Wiskott-Aldrich Syndrome protein is used to alleviate the pathologies associated with Wiskott-Aldrich Syndrome.
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