Development and functional validation of a gene editing approach in hematopoietic precursor cells for Wiskott-Aldrich Syndrome Ghent University
The Wiskott-Aldrich syndrome (WAS) is an X linked disease caused by a defect in the WAS gene.
Affected individuals suffer from recurrent infections, eczema, bleeding, auto-immune symptoms
and an increased risk of leukemia. Since WAS protein is involved in the signal transfer from the T
cell receptor to the cytoplasm, the lack of this protein will result in a suboptimal immune
responses to pathogens. In addition, T cell ...