Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy


We have developed a producer cell line that generates lentiviral vector particles of high titer. The vector encodes the Wiskott-Aldrich syndrome (WAS) protein. An insulator element has been added to the long terminal repeats of the integrated vector to limit proto-oncogene activation. The vector provides high-level, stable expression of WAS protein in… (More)
DOI: 10.1038/mtm.2014.63


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