HAEMATOPOIETIC STEM CELL-GENE THERAPY FOR WISKOTT-ALDRICH SYNDROME

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.