Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome.

Although this did not reach statistical significance, WAS HSPC in vitro-derived platelets displayed decreased size and granularity compared to healthy controls and knock-in of the W-pA cassette in WAS cells rescued the phenotype (Fig. 3f, g ; Supplementary Fig. 9 ).

also in 111,027 other papers