Data stemming from our proof-of-concept study of GE applied to WAS hinted to full reconstitution of WASp expression contributing to more reliable correction of thrombocytopenia; if confirmed by further in vivo studies, this evidence could enable a broader application of GE also to patients with attenuated WAS who predominantly manifest thrombocytopenia and bleeding risk and who are excluded from GT applications because of limited benefits. This evidence concerns the gene WAS and Thrombocytopenia.