Recapture Lysosomal Enzyme Deficiency via Targeted Gene Disruption in the Human Near-Haploid Cell Line HAP1.

At a dose of 50 ng, we observed elevated levels of GBA (~25% of HAP1 parental), although this increase did not reach statistical significance as compared to those of non-treated KO controls ( Figure 3 ).

also in 111,027 other papers